News

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…

Jordan McLinn is turning 6 years old and his mom has a single birthday wish: the possibility of watching him grow up and have the opportunity of becoming the firefighter that he dreams of becoming. Jordan suffers from a deadly type of muscular dystrophy, Duchenne, which will begin to immobilize him within a few years and dramatically…

BioMarin Pharmaceutical Inc. announced the presentation of data regarding disease progression in patients with Duchenne Muscular dystrophy (DMD) at the 67th American Academy of Neurology (AAN) 2015 Annual Meeting in Washington, D.C., April 18-25. The presentation was entitled “Evaluating the Progression of Physical Impairment, Activity Limitation, and Quality…

The Indoor Waterpark and opening weekend at Camelback Lodge in Pennsylvania played a major role in making this year’s 28th annual MDA Ride for Life event a major success. The event, which was held between May 1 to 3, raised over $1.1 million to help families that are affected by muscular dystrophy and…

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 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…

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…

Researchers at the University of California San Diego recently published in the journal BMC Research Notes an approach to assess possible dysregulated mechanisms linked to Duchenne muscular dystrophy. The study is entitled “Dysregulated mechanisms underlying Duchenne muscular dystrophy from co-expression network preservation analysis.”…

The Muscular Dystrophy Association (MDA) announced May 1 that after careful consideration and analysis of evolving realities pertaining to both television viewing habits and to philanthropic giving, it is time for the organization to discontinue producing and broadcasting the MDA telethon after 58 years on the air, and move…

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…