CureDuchenne Blog

PTC Therapeutics Patient/Parent Call

  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 […]

Rare Disease Day

  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 […]

Update on Phase 3 Tadalafil Trial

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 […]

BioMarin DMD Update for Patient Groups

  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 […]

CureDuchenne-Funded Trial for Cardiac Therapy

  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 […]