Development of Drisapersen and Follow-On Compounds Discontinued

BioMarin Pharmaceutical announced May 31 that it is discontinuing development of its exon skipping drug drisapersen. The company recently withdrew its Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting that indicated the CHMP intended to issue a negative . . .

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Five Questions with ALS Researcher Kim Staats

Kim Staats, a postdoctoral researcher at the University of Southern California in Los Angeles, was awarded an MDA development grant totaling $180,000 over a period of three years to study potential causes for sporadic amyotrophic lateral sclerosis (ALS). Using a cutting-edge approach to identify genetic contributors in sporadic ALS, Staats has found a new gene . . .

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Santhera’s Congenital Muscular Dystrophies Drug Omigapil Receives FDA Fast Track Designation

    The investigational drug omigapil, under development by Santhera Pharmaceuticals, has received U.S. Food and Drug Administration (FDA) Fast Track designation for the treatment of congenital muscular dystrophy (CMD). Preclinical studies in disease models have shown the drug inhibits cell death and reduces body weight loss and skeletal deformation while increasing mobility and improving . . .

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Five Questions with ALS Researcher Kathrin Meyer

Kathrin Meyer, a postdoctoral researcher at Nationwide Children’s Hospital in Columbus, Ohio, was awarded an MDA development grant totaling $180,000 over three years to study the roles of cell types other than motor neurons in amyotrophic lateral sclerosis (ALS). Please describe your current research. For a long time, ALS research focused mainly on the motor . . .

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Five Questions with ALS Researcher Evangelos Kiskinis

Evangelos Kiskinis, assistant professor in the department of neurology & physiology at Feinberg School of Medicine, Northwestern School of Medicine in Chicago, was awarded an MDA research grant totaling $300,000 over three years to decipher the degree of mechanistic overlap in different forms of amyotrophic lateral sclerosis (ALS). Using cutting-edge technology, Kiskinis will activate ALS . . .

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MDA Appeals to FDA in Support of DMD Drug Eteplirsen

Dear MDA family and friends, As part of MDA’s commitment to provide our DMD community with up-to-date research and advocacy news, we want to let you know that MDA today made another appeal to the U.S. Food and Drug Administration in support of eteplirsen, which, if approved, could help slow progression of the disease for . . .

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Five Questions with ALS Researcher Claudio Hetz

Claudio Hetz, a professor in the Faculty of Medicine at the University of Chile, was awarded an MDA research grant totaling $294,000 over a period of three years to study a potential new therapy aimed at rescuing motor neurons in amyotrophic lateral sclerosis (ALS). In ALS, some proteins do not fold into their required shape . . .

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PLEO-CMT Trial Seeking Participants

Researchers are looking for people with type 1A Charcot-Marie-Tooth disease (CMT1A) to participate in a new pivotal phase 3 clinical trial of the investigational drug PXT3003. The trial aims to determine whether PXT3003 is effective and well-tolerated in people with CMT1A. PXT3003 is an oral low-dose combination of three different drugs designed to target the . . .

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Ranolazine Trial Seeks Participants with Paramyotonia Congenita

Researchers at The Ohio State University are looking for adults, ages 18 years or older, with paramyotonia congenita to participate in a phase 2 open-label study being sponsored by Gilead Science to test the experimental drug ranolazine. Ranolazine currently is approved by the U.S. Food and Drug Administration to treat chest pain in people with . . .

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