Articles

BioMarin Announces FDA Accepts Drisapersen NDA for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

June 29, 2015

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the U.S. Food and Drug Administration (FDA) has accepted for review the submission of a New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping,

Read more

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.

Read more

Akashi Reports Positive Clinical Data on HT-100

June 18, 2015

Akashi Therapeutics Inc., a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD), today announced positive interim clinical data from an ongoing Phase 1b/2a clinical program with HT-100 (delayed-release halofuginone) an orally available, small molecule developed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in boys with DMD.

Read more

Catabasis Pharmaceuticals Initiates MoveDMD a Phase 1/2 Trial of CAT-1004 for the Treatment of Duchenne Muscular Dystrophy

June 17, 2015

Catabasis Pharmaceuticals Inc. a clinical-stage drug development company built on a pathway pharmacology technology platform today announced that dosing of the first patient has been initiated in the MoveDMD trial a Phase 1/2 trial of CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD).

Read more

Santhera Update on Pre-NDA Meeting with FDA on Raxone®/Catena® in Duchenne Muscular Dystrophy (DMD)

June 10, 2015

Santhera Pharmaceuticals (SIX: SANN) announces that it has discussed its plans to submit a New Drug Application (NDA) for Raxone®/Catena® (idebenone) in DMD with the US FDA in a pre-NDA meeting.

Read more
Items 1 - 5 of 93  12345678910Next