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.
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.
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.
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.
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Translarnaâ„¢ (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.
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of RNA-based therapeutics today announced that it has initiated dosing in a confirmatory study of eteplirsen, the Company’s lead exon-skipping therapeutic candidate for the treatment of Duchenne muscular dystrophy (DMD), in ambulatory patients who meet specific criteria on their baseline 6-minute walk test score.
Capricor Therapeutics, Inc. (OTCBB:CAPR), a biotechnology company focused on developing novel therapeutics for the treatment of cardiovascular diseases, today announced positive data from the laboratory of Eduardo Marbán, M.D., Ph.D., Capricor’s Scientific Advisory Board Chairman and the Director of the Cedars-Sinai Heart Institute.
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of RNA-based therapeutics, today announced that it has initiated dosing in a clinical study of eteplirsen, the Company’s lead exon-skipping therapeutic candidate for the treatment of Duchenne muscular dystrophy (DMD), in patients who are non-ambulant or who have advanced DMD and don’t meet a minimum 6-minute walk test score at baseline.
