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Going Backwards to Go Forwards
I imagine that by now you have heard the news that BioMarin has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) in Europe. Like you, I am heartbroken and […]
The Wait for An Approved Drug for Duchenne Continues
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 […]
Sarepta Advisory Committee Meeting
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 […]
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 […]
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 […]
FDA Advisory Committee Meeting
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. […]
BioMarin Quarterly Update to 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 […]