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.
CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, will continue to watch the performance of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) during its acquisition of Prosensa, a biotechnology company CureDuchenne has supported at critical times.
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.
CureDuchenne has joined #GivingTuesday, a first of its kind effort that will harness the collective power of a unique blend of partners to transform how people participate in the giving season. Following the Thanksgiving holiday and the kickoff of the holiday shopping season, #GivingTuesday inspires people to give back to charities like CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy.
CureDuchenne is encouraged about BioMarin PharmaceuticalÌ¢âÂã¢s acquisition of Prosensa and their commitment to continue the development of drugs for Duchenne muscular dystrophy. BioMarin has a deep understanding of the orphan rare disease space, experience working with regulatory authorities worldwide and a successful record of getting drugs approved.
Duchenne Therapy Network and CureDuchenne released their newest video blog that highlights power wheelchair options available for Duchenne muscular dystrophy patients. Here we provide tips on what to look […]
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.
