The wait continues. Sarepta Therapeutics announced this morning that the FDA will not complete the review of the eteplirsen new drug application by the PDUFA date. Considering the thousands […]
As I prepare to travel to Monday’s Advisory Committee meeting I continue to be disappointed by the FDA Peripheral and Central Nervous System Drugs Advisory Committee’s initial review of […]
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 […]
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, 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 […]
We are looking forward to the U.S. Food & Drug Administration (FDA) Advisory Committee meeting on Tuesday, November 24. This will be an important day for the Duchenne community. […]
CureDuchenne is pleased to share the BioMarin Quarterly Update to the Duchenne Community. It covers important information about next week’s FDA Advisory Committee meeting and other key updates from […]
We are pleased to share the following letter from the CEO of PTC Therapeutics to the Duchenne community. CureDuchenne funded PTC Therapeutics back in 2003 and we are proud […]
