News

Sarepta Therapeutics To Submit Rolling New Drug Application For Duchenne Muscular Dystrophy Treatment Eteplirsen

Sarepta Therapeutics, Inc. , a biopharmaceutical company focused on developing innovative RNA-based therapeutics, has announced a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) pertaining to its lead product candidate eteplirsen — a Duchenne muscular dystrophy (DMD) treatment. Sarepta and the agency are agreed…

Catabasis Pharmaceuticals Will Present Webinar On The MoveDMD Trial

Catabasis Pharmaceuticals, a company focused on clinical-stage drug development that is supported by a pharmacology technology platform pathway, recently announced that Joanne Donovan, Chief Medical Officer, was invited by Parent Project Muscular Dystrophy (PPMD) to present the latest updates on the upcoming MoveDMD trial. MoveDMD is a Phase ½ clinical trial for …

La Jolla Acquires Rights To Next-Generation Gentamicin Derivatives To Advance Therapies For Duchenne’s

La Jolla Pharmaceutical Company, a drug development company working on therapies that address life-threatening diseases such as Duchenne muscular dystrophy, recently announced it has entered into an exclusive agreement to acquire the Indiana University Research and Technology Center’s (IURTC) intellectual property rights that cover the derivatives of their next-generation experimental…

Western Refining Launch Campaign to Help MDA Summer Camp

A chain of convenience stores has joined forces with the Muscular Dystrophy Association (MDA) to help improve the quality of life of pediatric patients suffering from muscular dystrophy. Over 260 Western Refining convenience stores have joined the campaign to raise funding to allow more children to participate in the MDA…

EspeRare Receives European Orphan Drug Designation for Rimeporide in Duchenne Muscular Dystrophy

EspeRare, a company focused on uncovering the potential of existing molecules to address severe therapeutic unmet needs in rare diseases, recently received European Medicines Agency (EMA) Orphan Drug Designation for their Rimeporide compound for the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a rare, inherited, life-threatening disorder that…