DART Therapeutics Continues Clinical Development of HT-100 for Duchenne Muscular Dystrophy

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DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), announced today that it has resumed clinical development for its lead drug candidate, HT-100 (delayed-release halofuginone) an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD.

PTC THERAPEUTICS RECEIVES POSITIVE OPINION FROM CHMP FOR TRANSLARNAâ„¢ (ATALUREN)

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PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the company's application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.

Summit Report Preliminary Results from Phase 1b Clinical Trial of SMT C1100 for DMD

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SMT C1100 is an oral small molecule utrophin modulator that has the potential to treat all patients with DMD, regardless of the underlying dystrophin fault causing the disease. The Phase 1b trial was a dose-escalating, open-label study conducted in pediatric patients with DMD to evaluate safety, tolerability and drug exposure.

Summit Report Preliminary Results from Phase 1b Clinical Trial of SMT C1100 for DMD

Research Articles
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SMT C1100 is an oral small molecule utrophin modulator that has the potential to treat all patients with DMD, regardless of the underlying dystrophin fault causing the disease. The Phase 1b trial was a dose-escalating, open-label study conducted in pediatric patients with DMD to evaluate safety, tolerability and drug exposure.

Santhera Announces Successful Outcome of Phase III Study with Catena®/Raxone® in Duchenne Muscular Dystrophy

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Santhera Pharmaceuticals (SIX: SANN) announces today that its Phase III DELOS study of orally administered Catena®/Raxone® (INN: idebenone) in patients with Duchenne Muscular Dystrophy (DMD) met the primary endpoint and achieved its primary objective of delaying the loss of respiratory function compared to placebo.

Sarepta Therapeutics Announces Eteplirsen Demonstrates Stability on Pulmonary Function Tests through 120 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy

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Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced new pulmonary function data through Week 120 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results through more than two years of treatment showed stable pulmonary function in the Intent-to-Treat (ITT) study population (N=12). These data are consistent with previously reported 120-week clinical data showing a general stabilization of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT).

Prosensa Patient Webinar

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Dr. Giles Campion, Prosensa’s Chief Medical Officer (http://www.prosensa.eu/) presented an overview of the latest drisapersen results in this morning’s patient webinar. An analysis of the Phase III data suggests that administering the drug earlier in the disease and treating longer can delay the progression of DMD.

Prosensa Reports Initial Findings from the Further Clinical Data Analyses of Drisapersen for the Treatment of Duchenne Muscular Dystrophy

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Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet medical need, today announced initial findings from further analyses from the aggregate data from the clinical development program of drisapersen for the treatment of Duchenne Muscular Dystrophy (DMD).

Sarepta Therapeutics Announces Eteplirsen Demonstrates Continued Stability on Walking Test through 120 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy

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Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced data through Week 120 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD).
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