Articles

Sarepta Therapeutics Announces FDA Has Filed Eteplirsen NDA for the Potential Treatment of Duchenne Muscular Dystrophy for Patients Amenable to Exon 51 Skipping

August 25, 2015

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has filed the New Drug Application (NDA) for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Approximately 13% of people with Duchenne muscular dystrophy are estimated to have a mutation addressable by Eteplirsen/exon 51 skipping.

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Sarepta Therapeutics Receives Rare Pediatric Disease Designation From FDA for Eteplirsen for the Potential Treatment of Duchenne Muscular Dystrophy

August 24, 2015

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for eteplirsen, a potential treatment for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to skipping exon 51. The Rare Pediatric Disease Designation supplements the Orphan Drug Designation and Fast Track Status previously granted by the FDA for eteplirsen.

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BioMarin Receives Rare Pediatric Disease Designation From FDA for Drisapersen for the Potential Treatment of Duchenne Muscular Dystrophy

August 19, 2015

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for drisapersen, a potential treatment for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 51 skipping treatment. The FDA has previously granted drisapersen Orphan and Fast Track Status, Breakthrough Therapy Designation, as well as Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.

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FibroGen Receives FDA Clearance to Proceed with Clinical Study of FG-3019 in Duchenne Muscular Dystrophy

July 27, 2015

FibroGen, Inc. (Nasdaq:FGEN), a research-based biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has completed its review of the Company’s investigational new drug (IND) application for the study of FG3019 in patients with Duchenne muscular dystrophy (DMD), and clinical study may proceed. FG-3019 is a fully human monoclonal antibody that inhibits the activity of connective tissue growth factor (CTGF), a common mediator of fibrotic disease. FG-3019 is currently in Phase 2 clinical studies for idiopathic pulmonary fibrosis and pancreatic cancer

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Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

June 29, 2015

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for eteplirsen on June 26, 2015.

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