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…
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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…
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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…
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…
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