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 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 […]
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 […]
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, […]
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 […]
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 […]
Research and collaboration are bringing Canadians closer to a cure than ever before. However, many parents fear that access to treatment may come too late for their child. by Denise Deveau, Sep 6, 2019, > published on Maclean’s A community-based initiative led by Jesse’s Journey, in collaboration with Stand for Duchenne Canada. For […]
Notes from the “Living with DMD” web-doc Tecima Productions team. Our small production team travelled across Canada, meeting with families whose children have Duchenne Muscular Dystrophy (DMD). We met with Dakota (age 6), Anakin (age 11) and Carl (age 35) at critical moments in their journey. Our goal was to produce a Web-doc in three […]
Duchenne muscular dystrophy seen through the eyes of the families There are over 7,000 rare diseases in Canada. Duchenne muscular dystrophy (DMD) is one of them. DMD afflicts children, especially young boys, and sometimes girls. This degenerative muscle disease causes loss of the ability to walk, heart and respiratory failure, and usually leads to premature […]
Correcting nonsense mutations with CRISPR / Cas9? Would it be possible to correct nonsense mutations with CRISPR/Cas9? Some patients with DMD have a point mutation (nonsense) that leads either to the absence or to the abnormal function of dystrophin. The objective of Professor Jacques P. Tremblay’s team is to develop therapies for DMD due to […]
