Articles

Phase 2 Study in Boys 6 - <10 Years of Age with Duchenne Muscular Dystrophy

December 17, 2014

A multi-center clinical study evaluating the safety & tolerability, efficacy, pharmacokinetics (what the body does to a drug) and pharmacodynamics (what a drug does to a body) of the new investigational compound, PF-06252616, in approximately 105 boys diagnosed with Duchenne Muscular Dystrophy (DMD) who are able to walk and climb stairs. This study is designed to support future potential regulatory filings.

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Stem cells faulty in Duchenne muscular dystrophy, researchers find

December 17, 2014

In a mouse model of Duchenne muscular dystrophy, muscle stem cells express connective-tissue genes associated with fibrosis and muscle weakness, according to a new study.

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SUMMIT RECEIVES REGULATORY APPROVAL TO INITIATE A MODIFIED DIET CLINICAL TRIAL OF SMT C1100 IN DMD PATIENTS

December 11, 2014

Summit (AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy ('DMD') and C. difficile infection ('CDI'), announces that it has received approval from the UK Medicines and Healthcare products Regulatory Agency and the Ethics Review Committee to initiate a Phase 1b modified diet trial of SMT C1100.

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ARMGO Pharma and Servier Announce Advancement of Rycal ARM210/S48168 into Clinical Stage Program Targeting Duchenne Muscular Dystrophy

December 4, 2014

ARMGO Pharma and Servier today announced the successful completion of preclinical efficacy and IND/CTA enabling studies with ARM210/S48168, along with a formal decision to advance the program into early clinical development initially targeting treatment for patients with Duchenne Muscular Dystrophy (DMD), the most common and severe form of muscular dystrophy.

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PTC Therapeutics Announces Launch of Translarna™ (ataluren) in Germany

December 3, 2014

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Translarna™ (ataluren) is now commercially available to patients in Germany, the first country to launch in the European Union (EU), with first shipments expected to begin this week.

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