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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 […]
Capricor Therapeutics Announces First Patient Treated In HOPE-Duchenne Clinical Trial
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 […]
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 […]
CureDuchenne Urges Regulators to Approve Eteplirsen, Stresses the Importance of Multiple Drugs in the Pipeline
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 […]
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 […]
BioMarin Update to the Duchenne Community-November 25, 2015
BioMarin expresses its heartfelt appreciation to the Duchenne patient organizations, families, boys and young men living with Duchenne muscular dystrophy (Duchenne) who voiced their perspectives at the November 24th […]
CUREDUCHENNE RESPONDS TO FDA ADVISORY COMMITTEE MEETING ON DRISAPERSEN FOR DUCHENNE MUSCULAR DYSTROPHY
CureDuchenne, the California-based nonprofit organization dedicated to finding cures for Duchenne muscular dystrophy, released the following statement from its co-founder and CEO, Debra Miller, following the meeting of the Peripheral and […]