FDA Advisory Committee Discusses Clinical Data Package for BioMarin's Kyndrisa(TM) (drisapersen) for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

November 24, 2015

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.

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BioMarin Announces Data Analysis Demonstrating Consistent Efficacy of Kyndrisa™ (drisapersen) in Comparable Patients Across Three Randomized Studies

November 20, 2015

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.

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Sarepta Therapeutics Announces Publication of Positive Long-Term Safety and Efficacy Data for Eteplirsen in the Annals of Neurology

November 17, 2015

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.

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Duchenne muscular dystrophy is a stem cell disease

November 16, 2015

A new study from The Ottawa Hospital and the University of Ottawa is poised to completely change our understanding of Duchenne muscular dystrophy and pave the way for far more effective treatments.

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Santhera Reports New Data and Updates on Regulatory Filings for Raxone® (idebenone) in Duchenne Muscular Dystrophy (DMD)

November 11, 2015

Santhera Pharmaceuticals (SIX: SANN) announces that it has now completed comparative analyses of the respiratory outcomes for patients in its successful Phase III DELOS trial with data from a natural history DMD patient cohort collected by the Cooperative International Neuromuscular Research Group (CINRG).

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