Clustered regularly interspaced short palindromic repeat (CRISPR) has arisen as a frontrunner for efficient genome engineering. How-
ever, the potentially broad therapeutic implications are largely unexplored.
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 […]
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) met to discuss the data submitted to support the New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping.
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 […]
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced data analysis for Kyndrisa demonstrating consistent evidence of efficacy in comparable patients across three randomized, placebo-controlled studies that were conducted contemporaneously. Kyndrisa is currently under review with U.S. and European health authorities for the treatment of patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.
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 […]
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-based therapeutics, today announced that the Annals of Neurology published online [link to final article] positive efficacy and safety results from a Phase IIb long-term open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
