26 Nov
  • By Greg Freeman
  • Cause in

DYSTANCE 51, a phase 2/3 clinical trial of investigational suvodirsen in Duchenne muscular dystrophy

Suvodirsen (formerly known as WVE-210201) is an investigational stereopure oligonucleotide being developed as a potential treatment for boys with Duchenne muscular dystrophy with mutations amenable to exon 51 skipping. Wave Life Sciences is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating illnesses. Wave has initiated DYSTANCE 51, a global […]

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19 Nov
  • By Greg Freeman
  • Cause in

Update on the Vamorolone Program

We will provide an update on the scientific rationale for use of vamorolone, and share some results from our open-label Long Term Extension study. The very important pivotal clinical trial is now seeking participants; we will review information about this trial. ReveraGen was recently awarded a grant to study family and physician preferences regarding the […]

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14 Nov
  • By Greg Freeman
  • Cause in

The CureDuchenne Biobank Webinar

On Tuesday, November 12, 2019, from 4:00 PM to 4:30 PM, we learned about the CureDuchenne Biobank, which launched at our 2019 FUTURES national conference and will continue collecting samples at select CureDuchenne events around the country. Topics and their respective speakers, include: An Introduction to our CureDuchenne Biobank and why we created it | […]

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12 Sep
  • By Greg Freeman
  • Cause in

Free Webinar: Italfarmaco Givinostat Development Program Update

The webinar will review the Italfarmaco Givinostat development program for Duchenne and Becker Muscular Dystrophy. Content that will be covered, includes: Updates from the EPIDYS Study, a phase 3 clinical trial of Givinostat in ambulatory boys with DMD. Data from Italfarmaco’s long term studies of Givinostat in DMD populations. 30 minute Q & A. Speakers: […]

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11 Sep
  • By Greg Freeman
  • Cause in

Duchenne Clinical Trials & Drug Development

In rare diseases such as Duchenne, where the disease population is small, the drug development process can vary from that of more common diseases, and totally new treatment approaches such as gene therapies are emerging. Therefore, it is critical that families accurately understand the process and how they can help support—and possibly benefit from—the development […]

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04 Apr
  • By CureDuchenne User
  • Cause in

Edasalonexent in Development for the Treatment of Duchenne: Information on Three Clinical Trials

Join to learn about edasalonexent, a novel oral NF-kB inhibitor in development for the treatment of Duchenne muscular dystrophy, regardless of mutation type. This webinar will include: An update on the currently enrolling global Phase 3 PolarisDMD trial An introduction to the open-label extension GalaxyDMD trial Results from the Phase 2 MoveDMD trial Click here […]

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17 Dec
  • By CureDuchenne User
  • Cause in

Steroid Use in Duchenne: Expert Dialogue on Latest Data

Healthcare professionals, personal caregivers and family members of those with Duchenne muscular dystrophy gathered to learn about new data and best practices for steroid use in Duchenne at a webinar recorded on December 11. This free web broadcast featured a dialog between three physician experts and an experienced Duchenne parent/caregiver. Participants were able to ask […]

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20 Nov
  • By CureDuchenne User
  • Cause in

CureDuchenne Webinar with Avidity Biosciences

CureDuchenne Webinar with Avidity Biosciences, a privately held biotech company pioneering a new class of precision medicines based upon antibody-oligonucleotide conjugates, to brief the Duchenne community about Avidity’s research in pre-clinical development of potential therapies to treat patients with Duchenne muscular dystrophy (DMD).  The Webinar will feature Debra Miller, founder and CEO, CureDuchenne and Arthur […]

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13 Aug
  • By CureDuchenne User
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Recording available – DuchenneXchange.org Hosts Free Webinar to Showcase Features of Online Platform Designed to Unite, Support and Educate Duchenne Community

DuchenneXchange, the dedicated online platform built by the Duchenne community for the Duchenne community, hosted a free hour-long webinar for Duchenne community members to learn about the benefits of the DuchenneXchange. If you were unable to attend, please view the presentation using the following link. Click here to watch the recording of the webinar. You’ll be prompted to […]

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27 Jul
  • By CureDuchenne User
  • Cause in

Sarepta’s RNA-targeted technology platforms (PMO and PPMO)

Dr. Gilmore O’Neill, Chief Medical Officer of Sarepta Therapeutics, Inc, gave a scientific overview of Sarepta’s RNA-targeted exon skipping technologies, PMO and PPMO. Dr. O’Neill provided valuable information on this groundbreaking science. If you were unable to participate, please use the link below to view the recording. Click here to watch the recording of the webinar. You’ll be […]

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