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 -- If Approved, Kyndrisa Would Become the First Disease-Modifying Therapy for Patients With Duchenne Muscular Dystrophy in the United States Amenable to Exon 51 Skipping -- SAN RAFAEL, Calif.,...

BioMarin Posts Presentation From FDA Advisory Committee Meeting for Kyndrisa(TM) (drisapersen) for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping SAN RAFAEL, Calif., Nov. 24, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the Company’s presentation from the Peripheral and Central Nervous System...

Prosensa announces Lancet Neurology publication of an exploratory phase II study (DEMAND II) demonstrating efficacy and safety of drisapersen in patients with Duchenne muscular dystrophy Leiden, The Netherlands, Sept. 8, 2014 (GLOBE NEWSWIRE) -- LEIDEN, The Netherlands - September 8, 2014 - Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating...

Prosensa Completes Enrollment in Natural History Study of Duchenne Muscular Dystrophy Leiden, The Netherlands, June 25, 2014 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high...