15 Sep
  • By Alicia Clough
  • Cause in

Join CureDuchenne for a live Twitter chat with special guest Dr. Perry Shieh from the Center for Duchenne Muscular Dystrophy at UCLA

CureDuchenne is hosting a Twitter chat #DuchenneChat on September 20 at 11 a.m. PDT/2 p.m. EDT to raise awareness and address the important issues facing the Duchenne community. This Twitter chat will be an opportunity to hear from patients, parents, caregivers and will feature our special guest Perry Shieh, MD, practicing clinician at the Center […]

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11 Sep
  • By Alicia Clough
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CureDuchenne has Funded More than $1 Million for Dr. Flanigan’s Duplication 2 Research. Here’s the Latest Research Update

Dr. Kevin Flanigan from Nationwide Children’s Hospital provided an update on his Dup2 research during a CureDuchenne hosted call with families on September 6. Back in 2010, Dr. Flanigan told me he was working on duplication mutations.  We knew so many families with these rare mutations, and at that time, the excitement seemed to be […]

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27 Jun
  • By Alicia Clough
  • Cause in

Collaboration to Fund CRISPR/Cas9 for Duchenne

Duchenne organizations are invited to fund CRISPR/Cas9 research at Exonics Therapeutics in a venture philanthropy collaboration. We are making progress on treatments for Duchenne, but a cure is the goal our hearts long for. With the innovative CRISPR/Cas9 research being conducted by Dr. Eric Olson at the University of Texas Southwestern Medical Center, there is […]

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13 Jun
  • By Alicia Clough
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Beyond the Lab – CureDuchenne Ventures Helps Accelerate Academic Research into Promising Medical Care for Patients

Academia has long been an incubating source of incredible medical breakthroughs. Sometimes it takes a little push and risk-taking to take that progressive research out of the lab and into a business environment to achieve life-changing treatments for patients. Such is the case for a promising new technology that has the potential to help 80% […]

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09 Jun
  • By Alicia Clough
  • Cause in

Meet Alex Chiabai – First Boy in Canada to Begin Drisapersen Redosing

Five years ago, 12-year-old Alex Chiabai was one of the first boy in Canada to begin taking drisapersen, a promising drug that provided a glimmer of hope for Alex and his family. After stopping and starting the drug several times over the past few years, Alex once again has the distinction of being the first […]

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06 Jun
  • By Alicia Clough
  • Cause in

PTC /Ataluren – FDA AdCom Meeting September 28, 2017

CureDuchenne is pleased to see that the FDA will hold an Advisory Committee Meeting (AdCom) for PTC Therapeutics’s drug Ataluren.  We are hopeful for a full and fair review which will prioritize the patient and physician voice and experience. Rare diseases are complicated and regulators need to hear about the real world benefit that patients […]

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01 Jun
  • By Alicia Clough
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CD Access Began Redosing Drisapersen in Canada

For Doug Penner, now 14, being part of the original drisapersen trial was a hopeful time; the drug enabled him to go on four-mile walks, climb stairs and even jump. For a 9-year old boy with Duchenne this was remarkable. For Doug’s parents this was a time of enormous hope. Created by Prosena, drisapersen was […]

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17 May
  • By Alicia Clough
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Groundbreaking Research on Gene Editing for Duchenne Presented at ASGCT

The 2017 American Society of Gene & Cell Therapy took place in Washington DC last week. The Duchenne community was treated to a wealth of data from projects utilizing gene editing and gene therapy to treat Duchenne muscular dystrophy. On Wednesday, May 10, the well-attended session entitled “Gene Therapies for Musculoskeletal Diseases was chaired by […]

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16 May
  • By Alicia Clough
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EMFLAZA Bridge Program and Prescription Start Form

PTC is committed to getting patients onto EMFLAZA as quickly as possible, without interruption of therapy. For patients that are currently accessing EMFLAZA through Master Pharmacy importation from outside of the US, they may have immediate access to a “Bridge Program” that will provide EMFLAZA free of charge while their insurance status is being investigated. […]

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11 May
  • By Alicia Clough
  • Cause in

Dr. Olson Presents on Gene Editing

Today, Dr. Eric Olson, PhD, Chair of Molecular Biology at UT Southwestern, was honored to give a presentation at the American Society of Gene & Cell Therapy (ASGCT) 2017 meeting in Washington DC for the George Stamatoyannopoulos Lecture series. Dr. Olson presented new research showing how gene editing technologies like CRISPR/Cas9 and Cpf-1 can be […]

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