COVID-19 Resources

CureDuchenne is on this journey with you

A Message from Debra Miller | Founder & CEO of CureDuchenne

As a mom, a family, a friend, an advocate and a community, we know that the spread of the coronavirus disease 2019 (COVID-19) is clearly creating challenging times that call for the entire Duchenne community to come together and support each other. Through these challenging times, I wanted to personally assure you that CureDuchenne is here on this journey with you.

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We promise to continue to commit ourselves to providing the Duchenne community the most timely and accurate information we can, drawing from the relationships and resources CureDuchenne has built over the past 17 years.

CureDuchenne is here, still committed to helping every individual with Duchenne, their families and communities. We know that the coronavirus is presenting special circumstances for everyone, and especially for people living with Duchenne. We will provide information, solutions and advice on our website and social media channels for the unique needs of the Duchenne community.

CureDuchenne Webinar

March Updates on COVID-19 Vaccines – What the Duchenne Community Needs to Know

This webinar will be a roundtable with scientific and medical experts covering recent updates to COVID-19 vaccines and how they relate to the Duchenne community.

COVID-19 Vaccine Basics

There are many vaccine candidates in development, but currently there are three that have been authorized by the FDA for Emergency Use Authorization in the US:

  • Pfizer-BioNTech COVID-19 Vaccine, developed by Pfizer and BioNTech
  • Moderna COVID-19 Vaccine, developed by Moderna in partnership with the NIH, National Institute of Allergy and Infectious Diseases (NIAID)
  • Johnson & Johnson (J&J)/Janssen COVID-19 Vaccine, developed Janssen, a pharmaceutical arm of J&J, in collaboration with Beth Israel Deaconess Medical Center
  Pfizer–BioNTech
COVID-19 Vaccine
Moderna
COVID-19 Vaccine
J&J/Janssen
COVID-19 Vaccine
Vaccine type mRNA vaccine mRNA vaccine Adenovirus-delivered DNA vaccine
Doses required 2 (3 weeks between shots) 2 (4 weeks between shots) 1
Shipment and storage requirements Must be kept ultracold, -94 degrees Fahrenheit; once thawed must be refrigerated and used within 5 days Must be kept at -4 degrees Fahrenheit; once thawed must be refrigerated for one month Can be refrigerated for up to 3 months at 36-46 degrees Fahrenheit
Reported efficacy 95% effective seven days after the second dose 94% effective fourteen days after the second dose 66% effective against moderate to severe cases 28 days after single dose, 100% effective in preventing COVID-19–related hospitalization and death
FDA (US) Indication Intramuscular injection (shot) to individuals ages 16 and older to prevent COVID-19 infection Intramuscular injection (shot) to individuals ages 18 and older to prevent COVID-19 infection Intramuscular injection (shot) to individuals aged 18 and older to prevent COVID-19 infection
FDA Fact Sheet (includes ingredient information) Download Here Download Here Download Here
Product Website Visit Website Visit Website Visit Website

Last Updated: 3/17/2021

The CDC has a helpful explanation of how these COVID vaccines work, as well as information on the Emergency Use Authorization process, all of which can be found here.

Other vaccines that use different types of technology will likely come on the scene in the coming months.

Getting Vaccinated

Can I get vaccinated and, if so, where and when? What about carriers?

Vaccine administration has started in the United States for individuals ages 16 and older.

Due to limited vaccine supply, the CDC’s Advisory Committee on Immunization Practices (ACIP) is making recommendations for who should get vaccinated first. However, the specifics of who is eligible to receive a vaccination, and on what date, is being managed at the state or local level, and evolving all the time. Therefore, it will be most helpful to visit your state’s Department of Public Health website for the most up-to-date information.

That said, ACIP has recommended individuals ages 16-64 who are at a high risk for severe complications, as well as those who might be at high risk for severe complications, receive early vaccination under Phase-Ic of the roll-out. “Duchenne muscular dystrophy” is not currently listed as a high-risk condition, however, high-risk conditions (outlined here) include many conditions, some of which may be applicable to the Duchenne community: immunocompromised state due to corticosteroid use, obesity, and cardiomyopathy. CureDuchenne wants to remind the Duchenne community that cardiomyopathy is a condition which affects most individuals with Duchenne as well as many female carriers; this should be conveyed to providers when inquiring about vaccine eligibility.

When will children under age 16 get vaccinated?

Clinical trials of COVID-19 vaccines for children ages 12-17 have started in the US. Information about Pfizer-BioNTech’s clinical trial in children can be found here, Moderna’s here, and J&J/Janssen’s here. Additionally, Moderna’s trials for children aged 6 months to 12 years can be found here.

Dr. William Gruber, Senior VP of Vaccine Clinical Research and Development at Pfizer, notified the Duchenne community during CureDuchenne’s January COVID-19 Vaccine Webinar that enrollment in Pfizer’s Phase III study for 12-15 year olds was complete. He shared that Pfizer hopes to have the data submitted to the FDA for potential Emergency Use Authorization prior to the start of the 2021-2022 school year, though many steps remain before it can be widely administered in this age group.

Information on testing and availability of COVID-19 vaccines for children under the age of 12 remains to be determined.

If my neuromuscular specialist is out of state, can I get vaccinated at their clinic?

This may vary state-to-state.

CureDuchenne encourages you to check the relevant State Department of Public Health website for information about out-of-state resident travel restrictions (such as quarantine periods) and vaccine availability for out-of-state residents, or to contact your neuromuscular provider’s office.

Which vaccine can I/should I get?

This is, first and foremost, a conversation to have with your doctor or the healthcare provider administering your vaccine.

Individuals who are 16 or 17 years old are currently only eligible for the Pfizer-BioNTech COVID-19 Vaccine. If you are 18 years old or older, you are eligible for either the J&J/Janssen vaccine or the Moderna vaccine.

Other than those age restrictions, it is anticipated that many individuals will not have a choice of vaccine due to the impact of the limited supplies, especially in early phases of distribution. Public health officials are advising individuals to get whatever vaccine they have access to, as all vaccines available via Emergency Use Authorization (EUA) in the US have demonstrated significant benefit in reducing the frequency, severity and mortality of COVID-19 infections.

Is getting vaccinated safe?

Yes. The vast majority of clinicians and scientists agree that the drug development, review, and approval processes, as well as the information gained throughout the clinical trials for the COVID-19 vaccines, indicate that the vaccines are safe and highly effective. Trials run by all of the companies employed independent data safety monitoring boards which were tasked with reviewing safety data throughout the trials and prior to FDA submission.

While the development, testing and approval processes for COVID-19 vaccines were rapid, this was due, at least in part, to an unprecedented amount of collaboration between countries, agencies, drug companies, and scientists. Many drug companies also received financial support, through Operation Warp Speed and other avenues, allowing them to focus their efforts on COVID-19 vaccine development without financial setback. Recruitment to clinical trials also proved faster than normal, thanks to the amount of public awareness, and companies ran some of the testing processes in parallel to gather as much information as quickly as possible.

Additionally, mRNA vaccines have been under development since the early 1990s. BioNTech (who partnered with Pfizer) and Moderna, both pioneers in the mRNA field, have been able to use their existing platforms and technology to speed the development of their vaccines.

The J&J/Janssen vaccine comes out of decades of research on adenovirus-based vaccines. The adenovirus is used to deliver the coronavirus protein gene into cells, but the adenovirus has been modified so it cannot make you sick.

This graphic from the CDC helps explain the clinical trial and approval processes for vaccines, as well as three organizations and agencies tasked with monitoring vaccine safety once approved and being administered.

It is important to note, however, that all vaccines carry the risk of potential side effects. Pain and/or swelling at the injection site, fever, chills, fatigue, and headache are some of the most common. As referenced below, there is also a risk of allergic reaction with the vaccines. Its important to discuss these risks with your doctor.

Lastly, the available COVID-19 vaccines cannot give you COVID-19. This is because none of the vaccines use live coronavirus.

I heard that the vaccine can cause myalgia, or muscle ache. Is there a risk of rhabdomyolysis associated with being vaccinated?

Dr. Kathy Mathews, Professor of Pediatrics and Neurology at the University of Iowa, noted during CureDuchenne’s January COVID-19 Vaccine Webinar that the myalgia from vaccination is not typically associated with rhabdomyolysis. “I would not let that fear make you not get it; the risks of COVID[-19] far exceed any risk of muscle injury.”

I was previously diagnosed with COVID-19 or previously tested positive for antibodies. Should I still get vaccinated?

This is, first and foremost, a conversation to have with your doctor or the healthcare provider administering your vaccine.

The Centers for Disease Control and Prevention (CDC) says, “Due to the severe health risks associated with COVID-19 and the fact that re-infection with COVID-19 is possible, people may be advised to get a COVID-19 vaccine even if they have been sick with COVID-19 before.” This is also true for those who previously had positive antibody tests to COVID-19.

The CDC also says individuals should not be vaccinated against COVID-19 until the acute infectious period is over; those seeking vaccination who have had recent COVID-19 exposure should not do so until isolation/quarantine criteria are complete. Because it appears that immunity to COVID-19 lasts at least 90 days, individuals can (but need not) defer vaccination until 90 days from infection.

I’m on immunosuppressive medications. Is it safe for me to be vaccinated?

Pfizer-BioNTech’s and Moderna’s vaccines are approved for use in individuals who are immunocompromised due to corticosteroid usage. J&J/Janssen encourage individuals to speak with their provider.

The CDC classifies individuals on corticosteroids as being at a potentially increased risk for severe illness from the virus, enabling these individuals to be early recipients of the vaccine. Individuals on immunosuppressive medications were also included in the trials for these vaccines.

Dr. Anthony Fauci, the nation’s leading infectious disease expert and the director of the National Institute of Allergy and Infectious Diseases (NIAID), spoke at a medical conference in December 2020 where he suggested that individuals on immunosuppressive medications may not achieve the same level of immunity from the vaccine, but that any immunity is better than none.

The risk to immunocompromised individuals considering being vaccinated is limited to “live” vaccinations, and per the CDC, none of the vaccines approved for use or in development in the United States contain live virus.

I’m on immunosuppressive medications. Will they reduce my immune response to the vaccine, or otherwise limit the effectiveness of the vaccine?

During CureDuchenne’s January COVID-19 Vaccine Webinar, Dr. Hank Mayer, Director of the Pulmonary Function Lab at Children’s Hospital of Philadelphia, said, “There is a potential that the vaccine may not have the same immunogenic response that you would see in somebody NOT on chronic steroids. All we can do is hypothesize at this point because we just don’t have the data.” Dr. Mayer continued, saying, “The very robust tracking that’s being done should allow us to answer some of those questions before we get to the point where vaccination will be open to our patients with Duchenne muscular dystrophy.”

If you have questions about how to time vaccine administration with your steroid dosing, consult your neuromuscular specialist.

In addition to Duchenne, I’ve been diagnosed with an autoimmune condition. Should I still get vaccinated?

This is, first and foremost, a conversation to have with your doctor or the healthcare provider administering your vaccine.

At this time, there is no information to suggest that individuals with an autoimmune condition not get vaccinated. Adults with autoimmune conditions were eligible for enrollment in clinical trials. Additional information from the CDC can be found here.

Is there anyone who shouldn’t get vaccinated?

At this time, children under the age of 16 cannot/should not get vaccinated unless participating in a COVID-19 clinical trial.

Additionally, the CDC also says individuals should not be vaccinated against COVID-19 while they have an active COVID-19 infection, and instead should wait until the acute infectious period is over. Those seeking vaccination who have had recent COVID-19 exposure should not do so until isolation/quarantine criteria are complete. Because it appears that immunity to COVID-19 lasts at least 90 days, individuals can (but need not) defer vaccination until 90 days from infection.

Lastly, the CDC also has information available regarding allergy risk of the vaccines. Individuals who experience an allergic reaction to their first dose of COVID-19 vaccine, and those with a history of allergy to any of the vaccine’s components (such as polyethylene glycol, or PEG) should not be vaccinated. Individuals with a history of severe allergies (but not to components of the vaccine) should discuss with their doctor or vaccine provider prior to being vaccinated. For some individuals, being monitored closely in a provider’s office after administration may be sufficient.

I am currently enrolled in or will be enrolling in a clinical trial. Should I get vaccinated?

This is, first and foremost, a conversation to have with the clinical trial study team.

Each study sponsor will approach this differently, and in some cases, recent vaccination may affect your eligibility and/or timing of eligibility—for example, some trials do not allow for vaccinations within the 4-week period leading up to treatment.

Some considerations for clinical trial participants include potential drug-to-drug interaction between the vaccine and the investigational agent(s), ability to differentiate side effects of a vaccine from the investigational agent(s), and a vaccine’s role on an individual’s immune system. Because these answers vary based on the individual and the trial/investigational agent(s), do not proceed with vaccination until you have discussed with your clinical trial investigator or other study staff.

Will being vaccinated (or not being vaccinated) impact my ability to participate in clinical trials in the future?

This is, first and foremost, a conversation to have with the clinical trial study team.

Most exclusion criteria regarding vaccinations are short-term, meaning recent vaccinations (usually within a 4-week period) may affect an individual’s eligibility. Many trials also include instructions on what, if any, vaccinations can be administered once an individual is participating in a trial.

Few trial sponsors have yet released comprehensive information regarding COVID-19 vaccination affecting eligibility or participation, however Sarepta has posted a bulletin outlining their decisions.

Will being vaccinated affect my ability to participate in future gene therapy trials?

Many families are concerned that being vaccinated against COVID=19 may affect their ability to participate in future gene therapy trials due to potential impact on neutralizing antibodies. Sarepta posted a bulletin which clarifies information on mRNA vaccines and AAV gene therapies. A few important details from their bulletin:

  • There is currently no evidence to suggest that an mRNA-based COVID-19 vaccine (like Pfizer-BioNTech and Moderna’s) would cause an increase in neutralizing or total antibodies against adeno-associated virus (AAV). The antibodies an immunized individual would develop would be against this particular strain of coronavirus, not AAV—the two viruses are unrelated.
  • Both the J&J/Janssen vaccine and the Oxford-AstraZeneca vaccine candidate use adenovirus vectors. Adenovirus (AV) and adeno-associated virus (AAV) are not the same virus; there is currently no evidence to suggest that receiving an AV-based vaccine would affect total or neutralizing AAV antibodies.
  • Additional vaccines and treatments using AAV vectors are under development; these may affect an individual’s total or neutralizing antibody levels to AAV, which may impact eligibility to participate in future gene therapy trials. It is worth noting that currently, none of these projects are undergoing human clinical trials in the US. CureDuchenne will provide additional information as it becomes available.

In addition to checking with the study team, you can review the clinical trial information posted on clinicaltrials.gov and check our COVID-19 Duchenne Industry Announcements page for updates.

Understanding the Vaccine’s Role in the Pandemic

If I get vaccinated, does that provide any protection to others (including those I live with) who aren’t eligible for vaccination?

Yes, though maybe not immediately.

Think about the annual flu vaccine: the US makes a big push for as many Americans as possible to get vaccinated in order to reduce an individual’s risk of contracting the flu, but also in order to reduce the nationwide case count and to protect those who can’t get vaccinated. This is called herd immunity, a term used to describe a situation when the virus can no longer spread rapidly because of the number of people with immunity to it. (One other way to reach herd immunity is to let the virus run rampant through communities; some countries and municipalities, including Sweden, initially opted for this approach, though most have since abandoned it in favor of stricter precautions).

Experts aren’t sure what percentage of Americans or global citizens will need to be vaccinated against COVID-19 for herd immunity, or when we will reach this point. Given how contagious COVID-19 is, it is expected that about 70% of the national population will need to have been infected or vaccinated in order to reach herd immunity.

If people are getting vaccinated now, why are some communities still seeing high rates of COVID-19 infection

Simply put, vaccines don’t save lives—vaccinations do. The CDC said,as of March 4, 2021,about 54 million Americans of our nationwide total 328.2 million—roughly 16.3% of the national population—have received 1 or more doses of the available COVID-19 vaccines. (Only 8.4% of the population has received 2 doses).

I’ve been vaccinated. Do I need to wear a mask and/or socially distance?

Yes.

Although scientists need more time to determine whether those who have been vaccinated can be “silent spreaders” of COVID-19, in March, 2021 the CDC published  their Interim Public Health Recommendations for Fully Vaccinated People.

Fully vaccinated people can:

  • Visit with other fully vaccinated people indoors without wearing masks or physical distancing
  • Visit with unvaccinated people from a single household who are at low risk for severe COVID-19 disease indoors without wearing masks or physical distancing
  • Refrain from quarantine and testing following a known exposure if asymptomatic

For now, fully vaccinated people should continue to:

  • Take precautions in public like wearing a well-fitted mask and physical distancing
  • Wear masks, practice physical distancing, and adhere to other prevention measures when visiting with unvaccinated people who are at increased risk for severe COVID-19 disease or who have an unvaccinated household member who is at increased risk for severe COVID-19 disease
  • Wear masks, maintain physical distance, and practice other prevention measures when visiting with unvaccinated people from multiple households
  • Avoid medium- and large-sized in-person gatherings
  • Get tested if experiencing COVID-19 symptoms
  • Follow guidance issued by individual employers

Follow CDC and health department travel requirements and recommendations

Will being vaccinated impact my ability to attend school, fly, eat out, etc.?

Individuals who get vaccinated receive a COVID-19 vaccination card, with proof of vaccination. At the moment, these are used to help ensure individuals receive both doses of their vaccine at the correct intervals, and to help with tracking of administration.

Historically, some countries have used immunity passports to limit travelers to only those with vaccination history against diseases like Polio. The current vaccination card being supplied by the CDC is not the same as an immunity passport.

It remains unclear whether countries, airlines, states, schools, or companies will limit activities based on COVID-19 vaccination status.

Have a question?

Duchenne Industry and Partner Announcements

Astellas Pharma

Contact

Website: https://newsroom.astellas.us/covid19response
General Questions: Lauren Hicks, CPXP lauren.hicks@astellas.com
Medical and/or Clinical Questions: 1-800-727-7003
09/23/2020
Update:

Astellas’ Efforts Against the Spread of the Coronavirus Disease (COVID-19)


03/25/2020
Update:

COVID-19 Supply Situation

Audentes

03/24/2020
Update:

Community update from Audentes

Cumberland Pharmaceuticals

Contact

Website: http://www.cumberlandpharma.com/
Phone: 1-201-986-3860
Email: Ines Macias-Perez: imaciasperez@cumberlandpharma.com
08/27/2020
Update:

Mid-Year Check in and COVID-19 Updates


03/24/2020
Update:

Update from Cumberland Pharmaceuticals - As the coronavirus (COVID-19) continues to spread, our primary concern at Cumberland remains the health and safety of our associates, research partners and research subjects.

Italfarmaco

Contact

Website: https://www.italfarmaco.com/en-us/
E-mail: info@italfarmaco.com
For information regarding our clinical trials: patientadvocacy@italfarmaco.com; achristensen@trinds.com
For general inquiries: info@italfarmaco.com
08/04/2020
Update:

Stay up to date on Italfaramco’s Duchenne muscular dystrophy clinical trials here


03/25/2020
Update:

Message to families regarding COVID-19: CLINICAL TRIALS

NS Pharma

Contact

Website: http://www.nspharma.com/
Phone: 1-201-986-3860
E-mail: info@nspharma.com
For general inquiries regarding NS-065/NCNP-01 DMD clinical trial: trialinfo@nspharma.com
For DMD patients and families with research questions, email: DMDresearch@nspharma.com
03/23/2020
Update:

NS Pharma letter to the Duchenne Community on COVID-19

Pfizer

Contact

Website: https://www.pfizer.com/contact
Phone: 212-733-2323

Pfizer’s efforts to develop a COVID-19 vaccine are progressing with early positive data and they’ve dosed the first patients in the US as part of their global COVID-19 vaccine development program. See news about all their vaccine effort updates, along with other coronavirus education and resources including segments on:

  • How the immune system protects you from infection
  • Understanding social distancing: how far is enough?
  • Pfizer’s Chief Medical Officer, Dr. Mace Rothenberg’s videos on self quarantining, flattening the curve and tips to stay safe

PTC Therapeutics

Contact

Website: https://www.ptcbio.com/
PTC Cares: 1-844-478-2227
03/27/2020
Update:

Update on the Availability of EMFLAZA: PTC Therapeutics assures the Duchenne Community that they have more than a one-year supply of EMFLAZA in stock and available for delivery.


03/16/2020
Update:

Information from PTC Therapeutics on EMFLAZA Availability During Coronavirus (COVID-19) Crisis

ReveraGen BioPharma

Contact

Website: https://www.reveragen.com/
Email: suzanne.gaglianone@reveragen.com
Phone: 1-240-672-0295
3/23/2020
Update:

Statement to the Duchenne Community

Sarepta Therapeutics

Contact

Website: https://www.sarepta.com/
SareptAssist Patient Support: 1-888-727-3782

Addressing your questions about COVID-19 vaccination and gene therapy.

Solid Bio

Contact

Website: https://www.solidbio.com/
Email: Annie Ganot: annie@solidbio.com
03/27/2020
Update:

Letter to the Duchenne Community: COVID-19 Update

Updates from Government Agencies

Federal and State Coronavirus Information

Visit these sites for federal and state information about the coronavirus

Coronavirus (COVID-19) Website

For the latest from the combined efforts of the White House, the Centers for Disease Control and Department of Health & Human Services

The CDC also has some guidance for direct service providers, caregivers, parents, and people with developmental and behavioral disorders:

Centers for Medicare & Medicaid Services

FAQs on Prescription Drugs and the Coronavirus Disease 2019 (COVID-19) for Issuers Offering Health Insurance Coverage in the Individual and Small Group Markets

Food and Drug Administration (FDA)

The FDA is releasing COVID-19 related guidance on many topics to make regulations clear in support of coronavirus response efforts. The FDA has set up a website as a repository for all their latest information:

FDA Drug Shortage List
Drug Shortages can occur for many reasons, including manufacturing and quality problems, delays, and discontinuations. Manufacturers provide FDA most drug shortage information, and the agency works closely with them to prevent or reduce the impact of shortages.

Global collaboration on vaccine development
Peter Marks, MD, PhD, Director of the FDA Center for Biologics Evaluation and Research, typically the section of FDA where gene therapies are reviewed, wrote a blog about working globally to develop a vaccine for the coronavirus.


05/04/2020 Update: The FDA is optimistic that a COVID-19 vaccine will be approved within 9 months.

CureDuchenne attended an Axios webinar, sponsored by the Institute for Gene Therapies: Medical Research During a Crisis: Gene Therapy and the Future of Disease Treatment.

Peter Marks, MD, PhD, Director of FDA’s Center for Biologics Evaluation and Research, expressed continued optimism for gene therapies in spite of delays due to COVID-19. His division is also expecting to approve a COVID-19 vaccine in 9 months rather than the usual 12 to 18 months due to CBER’s efforts to “take away dead space in the development process.” While no part of the clinical trial process will be skipped, it’s important to find vaccines that are safe and effective fast in order to help keep all research on track. Dr. Marks believes that they’ll have a better understanding of the probability of success by mid-summer.

03/19/2020 Coronavirus (COVID-19) Update:
FDA Continues to Facilitate Development of Treatments

UK Based, Medicines and Healthcare products Regulatory Agency (MHRA)

03/20/2020 Update: Latest update from the UK government

National Institutes of Health (NIH)

View the latest update from the NIH, a part of the U.S. Department of Health and Human Services, the nation’s medical research agency.

World Health Organization (WHO)

Research Updates

PhRMA

PhRMA Alliance released several new COVID-19 tools:

  • This interactive clinical trials tracker features weekly updates on the progress the biopharmaceutical industry has made in beating COVID-19
  • This new glossary contains rollover definitions of common terms pertaining to COVID-19 therapeutics and vaccines

They also have several other new, useful resources available:

Additionally, plasma from those who have recovered could be a key part of the fight against COVID-19. If you’re interested in learning more about this and the plasma donation process, please visit TheFightIsInUs.org.

Other up-to-date resources on the biopharmaceutical industry’s efforts to combat COVID-19 are available on their coronavirus landing page.

Johns Hopkins University

CORONAVIRUS RESOURCE CENTER - Johns Hopkins experts in global public health, infectious disease, and emergency preparedness have been at the forefront of the international response to COVID-19.

This website is a resource to help advance the understanding of the virus, inform the public, and brief policymakers in order to guide a response, improve care, and save lives.

COVID-19 Treatment and Vaccine Tracker

The Milken Institute is engaged in efforts to help educate, inform, and support policymakers and businesses to:

  • Mitigate the spread of COVID-19.
  • Expedite the discovery and rollout of tests/treatments/cures.
  • Strategize on economic and policy levers to help companies weather the current conditions.

They have created a comprehensive online tracker that looks at all of the COVID-19 research currently in development and contains an aggregation of publicly-available information from validated sources. It is not an endorsement of one approach or treatment over another, but simply a list of all treatments and vaccines currently in development.

Institute for Health Metrics and Evaluation

This model gives a daily prediction of hospital capacity needs (# of beds, ICU occupancy, and ventilator use) and # of deaths due to COVID-19 for the next 4 months for each state in the US. Their predictions assume that every state will put social distancing measures in place within the next week, even those that haven’t yet done so.

Other Organizations

American College of Cardiology

03/16/2020
Update:

The current advice from the professional societies is to continue with ACE-I and ARBS.

Global Genes

03/23/2020
Update:

Global Genes Resource Guide

You've got questions, Global Genes has answers and wants you to know, you're not alone. Find your COVID-19 resources here.

Kaiser Family Foundation

03/26/2020
Update:

State Data and Policy Actions to Address Coronavirus

To date, states have taken a number of actions to mitigate the spread of the virus and reduce barriers to testing and treatment for those affected.

National Organization of Rare Disorders

03/05/2020
Update:

NORD’s Peter L. Saltonstall on Coronavirus Prevention and Risk for the Rare Community

Webinar - CureDuchenne Presents: Information, Updates and Mental Health Education

In this webinar, moderated by Tiffany Cook, MS, CCC-SLP, Senior Director of CureDuchenne Cares, the mental health presentation by Paige Lembeck, PhD, will be followed by several presentations from biopharmaceutical companies about ongoing clinical trials. We invite you to submit questions before and during the webinar to be addressed during a closing Q&A session.

Remember to keep proper nutrition when staying at home for long periods of time

Nutrition is the act or process of nourishing or being nourished. Research has indicated that proper nutrition is one component that can improve the overall quality of life for a person living with Duchenne muscular dystrophy. While there are several considerations regarding caloric consumption, including: physical activity, capability of ambulation, and steroid use, specialists in the field suggest that daily caloric consumption should be 80% of what individuals without Duchenne muscular dystrophy consume. Avoidance of excessive intake overall, salt intake not more than 1500 mg daily, and adequate calcium intake are recommended.

DuchenneXchange

DuchenneXchange was created with the vision of empowering those impacted by Duchenne through facilitating access to trusted information and support from the Duchenne community.

This innovative digital education platform was developed in collaboration with the Duchenne community, including our Curation and Engagement Councils, as well as members of CureDuchenne and rareLife solutions. Building off of rareLife’s “onevoice” community building technology, the collaboration members guided the customization of the platform by identifying knowledge gaps, sharing information preferences, and continually engaging their networks for additional insights.

A Physical Therapist’s Recommendations on Maintaining Physical Health

Ambulatory and non-ambulatory exercise are important aspects for maintaining physical health. Consistent and frequent physical therapy (PT) is one of the few treatments, at this time, for those with Duchenne. It’s important through all stages and ages of Duchenne, with the potential to delay progression of the disease.

In the absence of professional PT, you can find some guidelines on stretching exercises here:

These videos can help you keep moving when staying put is the mandate.

Managing anxiety is important to your mental state

“Tips on how ‘Managing anxiety is important to your mental state’, from Missy Dixon, PhD, MS, University of Utah, Pediatric Neurology and Pediatric Behavioral Health and Primary Children’s Hospital.

Cognitive Behavioral Strategies to Address Anxiety

Counterstimulation

  • Senses: Taste, Touch, Sight, Hearing, Smell
  • Simple, Strong, Pleasant
  • Light and relaxing

Distraction

  • Keep mind from focusing on worry and helps to relax
  • Activities, opposite emotions, pushing away, other options

Cognitive Control

  • Imagery
  • Mindfulness
  • Positive Self-talk

Relaxation

  • Breathing exercises: deep, paced
  • Progressive muscle relaxation
  • Mini relaxation
  • Apps and YouTube

PRACTICE! PRACTICE! PRACTICE!

Pacing techniques

  • Pace into change
  • Cannot go from 0 to 60 overnight

Set Alarms as reminders

Schedule fun and pleasant events

  • Don’t forget to do the fun stuff!

Rehearsing problem solving

  • Have a plan
  • Anxiety does not like a plan!

Positive Messages

  • Reminders of positive self-worth
  • Use sticky notes or any other ways to remind yourself

Build and communicate with your support network

Mental health & coping mechanisms

The sudden and near-constant stream of news reports about an outbreak can cause anyone to feel worried. Get the facts, not rumors and misinformation. Facts can help to minimize fears.

  • Seek information only from trusted sources.
  • Limit the news, and be careful what you read.
  • Gather information at regular intervals, once or twice a day, from the WHO and local health authorities.
  • Minimize watching, reading or listening to news that causes you to feel anxious or distressed.
  • Take practical steps to prepare your plans and protect yourself and loved ones.

Learn more from the World Health Organization

Coronavirus: How to protect your mental health

Coronavirus has plunged the world into uncertainty and the constant news about the pandemic can feel relentless. All of this is taking its toll on people's mental health, particularly those already living with conditions like anxiety and OCD. So how can we protect our mental health?

Being concerned about the news is understandable, but for many people it can make existing mental health problems worse.

When the World Health Organization released advice on protecting your mental health during the coronavirus outbreak, it was welcomed on social media.

Social distancing struggles?

Our Odyssey is hosting online meet ups to help with social and emotional support for young adults living with rare and/or chronic conditions during this time of increased isolation. This organization is a new NORD Gold Member and was founded by young adults living with rare and chronic conditions. Details are below; please share with your community:

Mental health support

We understand the importance of treating mental health in addition to physical health, as many patients are now isolated and concerned about what's to come. Here are some resources you can share with your community when they need additional support:

  • Mental Health America: dedicated to those living with mental illness and to promoting overall mental health
  • Center for Chronic Illness: provides educational and emotional support for people with rare/chronic illnesses via web-based support groups
  • Happy- 24/7: offers support and encouragement from peer support counseling (new users can try it free of charge)

Child neurology guidance during COVID-19 pandemic

For the child neurology community, where children and families may need to spend time in clinics and hospitals or may experience lowered immunity, we are sharing additional guidance for how to proceed during this time when COVID-19 has reached pandemic status. The Child Neurology Foundation (CNF) has worked in collaboration with the Child Neurology Society to release a joint statement on where to find the most up-to-date information, as well as some data on the effects of COVID-19 in the child neurology community and tips for staying safe.

Who should I call if I think my child is experiencing symptoms of COVID-19?

If you think your child may have COVID-19, call your primary care provider (PCP) for guidance. If your child is experiencing trouble breathing, call 911. It is important to get emergency help as quickly as possible, no matter what. Make sure you let the medics and staff know that your child has respiratory symptoms. If you know that your child has been exposed to someone who has COVID-19, share that information right away too.

Dr. Wong’s guide for Exon to staying healthy

Dr. Wong’s advice to Exon, the self-appointed mascot of the Duchenne community and patients, Dr Wong is the Founding Director of the Duchenne Program at University of Massachusetts Memorial Health Care

Covid19 Infographic
View the Infographic

The importance of physical therapy

No two patients are alike and Duchenne is a progressive condition that changes often. Duchenne is a very unique diagnosis and requires physical therapists to adopt a completely different frame of mind from their approach with other kinds of patients. Learn how to get the proper care for a Duchenne patient here.

Connect with a Physical Therapist on Facebook

CureDuchenne Certified Physical Therapists will be offering online support to families affected by Duchenne muscular dystrophy. Please post questions. We will moderate and respond as they come in.

Extensive list of mental health and education resources

Sara Turner, from Behavioral Health at Intermountain Primary Children's Hospital, put together an extensive list of mental health and education resources for parents and kids during COVID-19 and beyond. The information is not exclusive to Duchenne patient care but includes general coping and self-care, as well as ideas for keeping kids busy, learning, and engaged. We've found this resource to be super helpful and hope that you do too.

Do you or does your family member or patient have breathing muscle weakness for any reason?

If you have generalized muscle weakness, decreased breathing capacity, and a weak cough, then now is time to ASK YOUR DOCTOR about how to prevent respiratory failure and avoid invasive tubes. And if your doctor does not know about the benefits of noninvasive respiratory care, then today is the day to SHARE THIS INFORMATION with your physician.

Duchenne Family Assistance Program

If you need help with insurance claims and denials, coverage for approved treatments, or direct financial assistance for appointments, equipment or modifications, the Duchenne Family Assistance Program may provide some options.

04/10/2020 Update: Duchenne Family Assistance Program is launching the Coronavirus Relief Fund for Duchenne families.

Medicare broadened coverage for telehealth services

If you or your child is covered by Medicare - Starting on March 6th, Medicare broadened coverage for telehealth services. You may be able to access your physician and other providers by phone rather than having to risk traveling to a healthcare facility. It covers telehealth visits with doctors, nurse practitioners, clinical psychologists, and licensed clinical social workers.

Since this is newly in place, the best first step is to contact your provider to see if they can virtually meet with you.

For more details about Medicare telehealth services coverage:

National Organization of Rare Disorders

NORD has several assistance programs to help families with challenges arising from coronavirus:

  1. COVID-19 Critical Relief Program, 203.242.0497 or COVID19assistance@rarediseases.org
  2. Access Assistance Programs for DMD

    DMD specific resources:
    Duchenne Muscular Dystrophy | Accepting Applications
    Copay Assistance
    Medical Assistance
    Phone: 866-218-8172
    Email: DMD@rarediseases.org
    Fax: 203-263-9597

    Clinical Trial Travel & Lodging: PTC 124-GD-016-DMD
    Contact: 1-866-647-9325 x 333
    Email: PTC-016-DMD@rarediseases.org
    Fax: 1-203-674-9941

    Learn more about this program

    Clinical Trial Travel & Lodging: FG-3019
    Contact: 1-844-876-0774
    Email: fg-3019@rarediseases.org
    Fax: 1-203-798-7413

  3. Relief for Caregivers, 203.616.4328 or CaregiverRespite@rarediseases.org, https://rarediseases.org/for-patients-and-families/help-access-medications/patient-assistance-programs-2/#section-4

Resources for Preparing your Home, from the Centers for Disease Control (CDC)

Help to plan, prepare and respond to Coronavirus Disease 2019:

For more guidelines from the World Health Organization on this, start here

What you need on hand when remaining at home

It is wise to have extra of what you might use on a daily basis, as well as extra medical supplies. According to Muscular Dystrophy News, here are some items to be sure to include:

  • Medications like steroids
  • Feed for feeding tube users
  • Distilled water for BiPAP or CPAP machines
  • Bandages
  • Masks
  • Supplements
  • Rubbing alcohol
  • Syringes, vitamins
  • Laxatives
  • Stoma bags
  • Disinfectant wipes
  • Non- perishables such as: beans, soups, canned vegetables
  • Gatorade and other electrolyte drinks
  • Bread
  • Milk
  • Soap
  • Toothpaste

Here are some other Virtual Exploration & Educational Resources and activities to do with your family:

FOR AMBULATORY INDIVIDUALS

Top Tips from Physical Therapists

  1. Try to mimic normal level of activity as if you were on a regular day schedule
    • Walk around the block or the house as able without fatigue.
    • Best recommendation is to take multiple short trips throughout the day.
  2. Daily warm bath/shower, massage and stretching routine.
  3. Self-stretches throughout the day and movement breaks during home study, video games, tv watching
  4. Keep using AFOs on normal schedule: recommendation is 6-8 hrs per 24 hr period
  5. Add in other fine motor activities at home like art, cooking, games not just video games and iPads.

Exercises to help you at while at home:

Stretching
YouTube Default Photo

Printable Flyer

Self-Stretching
YouTube Default Photo

Printable Flyer

Massage
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Printable Flyer


Sample Daily Schedule:

Use this sample daily schedule to help you with provide a consistent amount of care. To help you stay organized, you can keep it color coded for type of activity: Stretch, Exercise, Rest

  • Stretch daily 30 min x 1-2 / day or as tolerated
  • Exercise daily 10 min x 4-5 /day or as tolerated
  • Rest every 20 min of video games or phone use. Prone rest 20 min 2 x/day no activity

FOR NON-AMBULATORY INDIVIDUALS

Top Tips from Physical Therapists

  1. AFOs 8 hrs/day
  2. Stretch routine 1-2 times per day; upper and lower extremity
  3. Lay prone 1-2 times per day for 20 minutes or more if able (use pillows to lessen pressure on abdomen)
  4. Ask school if stander can come home for this duration if there isn’t one at home for use
  5. Get outside for safe area walks and fresh air and sunshine
  6. Use power seating options for changing position and stretching
  7. Rest and stretch every 20 min of video games or phone use.

Exercises to help you at while at home:

Stretching
YouTube Default Photo
Massage
YouTube Default Photo
Pulley Exercises
YouTube Default Photo

Sample Daily Schedule:

Here is a sample daily schedule to help you with provide a consistent amount of care. To help you stay organized, you can keep it color coded for type of activity: Stretch, Exercise, Rest

  • Stretch daily 30 min x 1-2 / day or as tolerated
  • Powerchair stretching 20 min x 1-2 /day
  • Exercise daily 10 min x 3-4 /day or as tolerated
  • Prone rest 20 min 1-2 x/day no activity

School

I have concerns and questions about returning to school in the fall

Parents are understandably nervous about sending their kids back to school in the fall, especially because of increased risk due to Duchenne or related treatments. Here we have compiled some resources and answers to questions you may have:

What are the obligations of my local school district to provide distance learning alternatives and can they cite an inability to meet these requirements as a reason for closing the school?

Any educational opportunity offered to the student body or during a school closure must offer equal access to that education to everyone, and it is not a reason to close the school. This document from US Department of Education, Office for Civil Rights, Special Education and Rehabilitative Services has more information on this: https://www2.ed.gov/about/offices/list/ocr/frontpage/faq/rr/policyguidance/Supple%20Fact%20Sheet%203.21.20%20FINAL.pdf

Clinical

Have any recommendations, tips, etc. on general respiratory care?

“There is no specific modification to standard infection control (hand washing, staying away from sick people) that is needed. Aggresive airway clearance is needed, however, as with any acute illness. I don't have any documents specific to COVID-19 and Duchenne or neuromuscular disease, but would be happy to answer specific questions.”

Oscar Henry Mayer, MD
Medical Director, Pulmonary Function Testing Laboratory
Division of Pulmonology
The Children’s Hospital of Philadelphia

My son has a Trilogy BiPAP and is not showing any symptoms - but if necessary, can BiPAP can be turned into a ventilator?

“The Trilogy is a ventilator and can be used through a breathing tube (usually a tracheostomy tube) but is not designed for ventilation in unstable or very sick people. Having said that, it is certainly possible that ventilators like the trilogy may need to be used for sick patients in the hospital”

Oscar Henry Mayer, MD
Medical Director, Pulmonary Function Testing Laboratory
Division of Pulmonology
The Children’s Hospital of Philadelphia

We have not used cough assist yet. We have the machine settings set, and when not sick right now, wanted to find out how we use it?

“The cough assist is often used twice a day to help keep the airways clear and the lungs open. Then, every hour or so as needed during an illness. Individual specifics and plans should always be discussed with your healthcare provider before starting any new treatments. In these current conditions surrounding the spread of Coronavirus, individuals and caregivers are recommended to have these conversations as soon as possible to help prepare themselves.”

Oscar Henry Mayer, MD
Medical Director, Pulmonary Function Testing Laboratory
Division of Pulmonology
The Children’s Hospital of Philadelphia

How are Duchenne patients treated if they get Covid-19 since they can’t have straight oxygen?

“The key point to remember with the concern about COVID-19 is that while it is novel, which explains the relatively fast and broad distribution through the community, it behaves like other severe viruses that we are used to such as influenza, RSV, rhinovirus, and adenovirus. This is not to minimize COVID-19 or other viruses for that matter, but just to state the obvious. So the approach this year with a suspected respiratory viral illness is the same as in the past with the focus being on airway clearance. There remains no indication for “straight” oxygen unless the patient is first shown to not have respiratory failure. Once a patient is either shown to not have respiratory failure or it is treated well then supplemental oxygen is perfectly fine.”

Oscar Henry Mayer, MD
Medical Director, Pulmonary Function Testing Laboratory
Division of Pulmonology
The Children’s Hospital of Philadelphia

Clinical Trials

Does edasalonexent affect the immune system?

“Long-term toxicology studies with edasalonexent using higher doses than those in our clinical trials have found no evidence for immunosuppression using standard clinical and anatomic physiology methods. In clinical studies, now with over 100 patient-years of exposure to edasalonexent, we have found no evidence of immunosuppression or increased infections. In the Phase 3 PolarisDMD trial of edasalonexent, as well as the GalaxyDMD open-label trial, boys are not on steroids.”

Catabasis

Should trial participants still go to the hospital for their assessments?

“We are fortunate that site visits are relatively infrequent during the Phase 3 PolarisDMD trial with assessments every 3 months. Currently, we are focused on ensuring that patients have uninterrupted drug supply as well as safety monitoring. We are working closely with our clinical trial sites with frequent communication.”

Catabasis

Will the outcome of ongoing trials be endangered by not being able to carry them out as per protocol?

“We are actively monitoring the situation and have plans in place to address potential disruptions. Fortunately, we designed our clinical trial so that visits are relatively infrequent. We are working with sites to support drug supply, as well as safety and efficacy assessments.”

Catabasis

Medication

Although PTC has a large supply of EMFLAZA that will be unaffected by COVID-19, we are worried about running out. How can my family get more than a 2 week supply of EMFLAZA in our home?

Read PTC's full statement regarding EMFLAZA supply, shipping, and access here: http://ow.ly/AOPU50yXB2Q

“We note that while the supply of EMFLAZA that patients can order at one time can vary, many insurance companies are allowing flexibility in accessing treatment – including allowing patients to refill sooner to support their supply, and allowing prescriptions for a 90-day dispense of medication to ensure continual treatment. Patients are encouraged to work with their doctor to consider whether a 90-day supply is right for them. In the meantime, PTC is committed to ensuring continuity of treatment for every EMFLAZA patient should issues arise. For more information, patients can contact their PTC Cares team at 1-844-PTC-CARES (844-478-2227).”

PTC Therapeutics

Other (Duchenne-related)

In case the delivery person is infected/carrier/contagious, what precautions can we take to ensure mail and deliveries are virus free?

“According to the World Health Organization: "The likelihood of an infected person contaminating commercial goods is low and the risk of catching the virus that causes COVID-19 from a package that has been moved, travelled, and exposed to different conditions and temperature is also low.”

World Health Organization: https://www.who.int/news-room/q-a-detail/q-a-coronaviruses

Community Answers to Common Questions regarding Coronavirus and Duchenne

We are compiling FAQs from the community and will make them available here for quick answers to common questions. Use the form on this page to ask a question and we’ll help get you the answer.

Check back here and our Facebook page regularly for new answers to questions from throughout the community. Please note that not all questions can be answered, but we’ll do our best to answer as many as we can, as quickly as we can.”

Have a question?