Giving our Sons with Duchenne the Best Care Possible
When our son, Hawken, was diagnosed 13 years ago, we were told to go home and love your son…there is nothing you can do. Those doctors were wrong, […]
When our son, Hawken, was diagnosed 13 years ago, we were told to go home and love your son…there is nothing you can do. Those doctors were wrong, […]
PTC withdraws the regulatory filing in Canada so they can supplement the phase II data with the phase III, ACT-DMD data. Why is PTC withdrawing the NDS from Canada? […]
Stu Peltz from PTC Therapeutics hosted a call today regarding the Refuse to File letter from the FDA, dated February 22nd. More detailed information is available in the transcript […]
Today, the last day in February, is Rare Disease Day when the world pauses for a moment to focus on often overlooked diseases that affect relatively few people but […]
CureDuchenne is pleased to see our biotech partner Capricor Therapeutics progress with their HOPE-Duchenne clinical trial. Capricor announced today the treatment of the first patient with Duchenne muscular dystrophy-related […]
We are sharing the following information from Lilly on their Phase 3 Tadalafil Trial We are writing to share disappointing news about our Phase 3 study of tadalafil in […]
The Duchenne community continues its fight to get an approved treatment for Duchenne. The FDA released Briefing Document on eteplirsen, the drug for Duchenne muscular dystrophy submitted for approval […]
We wanted to share the following information from BioMarin in regards to yesterday’s news on the FDA decision on Kyndrisa (drisapersen). Dear Patient Group Representative, As you […]
Capricor Therapeutics is actively recruiting participants for a clinical trial for a potential cardiac cell therapy for those with Duchenne muscular dystrophy. CureDuchenne funded Capricor to support this trial […]