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Sarepta U.S. Patient Affairs – Newsletter for the Duchenne Community
View the Sarepta Summer 2019 Newsletter HERE.
Erin Frey to Lead CureDuchenne’s DMD Advocacy Efforts
The nonprofit CureDuchenne has chosen veteran advocacy leader Erin Frey to become its first director of Duchenne muscular dystrophy (DMD) advancement efforts. Read the full article HERE.
CureDuchenne Launches Website to Assist Newly Diagnosed Duchenne Families
NEWPORT BEACH, Calif. (August 26, 2019) – Receiving a positive diagnosis for Duchenne muscular dystrophy is devastating for new parents. Likewise, learning about the rare disease and how to care […]
There’s No Straight Line in Drug Development
Today’s FDA disapproval of Sarepta’s Vyondys 53 is disheartening, but a true example of just how difficult and costly the drug development process is. We appreciate Sarepta’s efforts and dedication […]
Catabasis Connection – Phase 3 PolarisDMD trial for edasalonexent
The latest edition of the Catabasis Connection newsletter discusses Phase 3 PolarisDMD trial for edasalonexent in Duchenne. Read the full newsletter here.
CureDuchenne Cares and Duchenne Family Assistance Program to Provide Support Services to Duchenne Community
Organizations to provide financial support for 50 Duchenne families to travel to Southern California for the CureDuchenne 2019 FUTURES national conference NEWPORT BEACH, Calif. (July 1, 2019) — CureDuchenne, the […]
Summary of Pfizer Phase 1 Microdystrophin Gene Therapy Trial
Pfizer made their first public disclosure on early data from their open-label Phase 1 microdystrophin gene therapy trial for the treatment of Duchenne. The program was obtained from Pfizer’s acquisition […]
Updates on CureDuchenne Gene Therapy Initiative
We are excited to report the progress of CureDuchenne’s Gene Therapy Initiative. From our investment, as early as 2010, in Nationwide Children’s permanent duplication2 skipping program, to what has become […]
Gene Therapy Initiative Accelerated with Vertex Acquisition of Exonics Therapeutics!
In 2003 when my son Hawken was diagnosed with Duchenne people were not contemplating a cure. To find a pharmaceutical company working on Duchenne was like finding a needle in […]