News

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…

In 2005, quarterback Vince Young led his teammates from the University of Texas to a coveted national title while the Revell family back in Austin was just discovering that their son suffered from Duchenne Muscular Dystrophy and would be confined to a wheelchair by the time he was in college. The Revell family’s story…

On May 2, 2015 Bill and Kim Procko will be hosting a skateboarding competition “Carve to CureDuchenne,” which is intended to benefit the nonprofit CureDuchenne organization that funds research projects hoping to find a cure for Duchenne muscular dystrophy and raises awareness about the disease. The fundraising event will take place at…

aTyr Pharma (“aTyr”), a biotherapeutics company involved in the discovery and clinical development of pioneering medicines for patients with severe rare diseases using its knowledge of Physiocrine biology, a newly discovered set of physiological modulator, recently announced that Resolaris has been granted Orphan Drug Designation by the U.S.

BioMarin Pharmaceutical Inc., a company that develops and commercializes biopharmaceuticals for serious diseases and medical conditions, recently announced conclusion of the continuing proposal of a New Drug Application (NDA) to the FDA for drisapersen, an investigational exon-skipping drug candidate aimed at treating the largest genetically defined subset of…