What’s next for edasalonexent in 2020?
La Force is happy to share the latest edition of the Catabasis Connection newsletter about the Phase 3 PolarisDMD trial enrolled in the expected patient population and their partnership with Duchenne UK to evaluate edasalonexent in non-ambulatory boys and men affected by Duchenne. A new Phase 2 non-ambulatory clinical trial in partnership with Duchenne UK! […]
Sarepta Therapeutics Announces Partnership with Roche
Sarepta Therapeutics Announces Partnership with Roche in Territories Outside the United States for its Investigational Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy, SRP-9001 Press release here Quick view Roche obtains the exclusive right to launch and commercialize SRP-9001 outside the United States At closing, Sarepta will receive an upfront payment of $1.15 billion, comprising $750 […]
FDA grants accelerated approval to Vyondys 53
Sarepta Therapeutics Announces FDA Approval of VYONDYS 53™ (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53 Source: Sarepta Therapeutics, Inc., Dec 12, 2019 – Read the original news here– The U.S. Food and Drug Administration granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne […]
DEFEAT DUCHENNE FAMILY FORUM – MONTREAL
We are excited to share that the next Defeat Duchenne Family Forum will take place in Montréal, Québec, on Saturday, May 2, 2020. Presented by PTC Therapeutics and in partnership with Jesse’s Journey, this unique educational event provides the opportunity for you – families and caregivers navigating the Duchenne journey, to come together with researchers, clinicians, and industry professionals for a day of education and inspiration. More than that, […]
The Power of Giving Back
GivingTuesday – the global day of giving Join us for the 7th annual GivingTuesday on December 3, 2019! GivingTuesday is a global day of giving that happens each year after Black Friday and Cyber Monday. It’s a time when Canadians, charities and businesses come together to celebrate giving and participate in activities that support charities and non-profits. There […]
A DMD gene therapy has been placed on clinical hold
Following the occurrence of a safety incident, the FDA has placed on hold the clinical trial for SGT-001, the Solid’s gene therapy candidate for Duchenne muscular dystrophy (DMD). This clinical hold is the second bad news the DMD community has received this month. Last week, Swiss pharma giant Roche announced it was terminating its study of […]
