Duchenne Muscular Dystrophy (DMD)

Sarepta Issues ‘Open Letter’ Explaining Development of Eteplirsen for DMD

In July 2012, Sarepta Therapeutics announced encouraging interim results for its phase 2b trial of eteplirsen, an experimental exon-skipping drug in development to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene.

MDA Applauds Congress for Including Two Neuromuscular Diseases in Pediatric Research Bill

In July, days before the U.S. House of Representatives and Senate went into recess, bipartisan legislation was introduced in both houses to strengthen research into pediatric diseases.

MDA Commits $10.7 Million to Neuromuscular Disease Research

The Muscular Dystrophy Association has awarded 33 new grants totaling $10,684,481 to fund research projects focused on uncovering the causes of, and developing therapies for, neuromuscular disease.

MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical Advisory Committees, and the grants took effect Aug. 1.

DMD Research Briefs: Exon Skipping Advances in Trials, Lab

Exon skipping is an experimental therapeutic strategy in which regions — exons— of a gene are targeted and blocked ("skipped") by laboratory-designed molecules. The goal is that the remaining genetic instructions will lead to production of a shorter but still-functional protein.

DMD/BMD — Daniel Michele, Ph.D.

Daniel Michele, associate professor in the department of molecular and integrative physiology at the University of Michigan in Ann Arbor, was awarded an MDA research grant totaling $364,965 over three years to study the underlying mechanisms of muscle weakness and muscle fatigue in Duchenne (DMD) and Becker (BMD) muscular dystrophies.

Survey Examines Newborn Screening for Neuromuscular Disease

MDA is conducting a survey designed to assess the experiences of parents in the United States whose babies underwent newborn screening at the time of birth, with an eye toward the future possibility of newborn screening tests being recommended for certain neuromuscular diseases in which therapy development is advancing rapidly.

PTC Update on the Development of Ataluren in Nonsense-Mutation DMD/BMD

New Jersey biopharmaceutical company PTC Therapeutics recently declared its intention to do whatever it takes — including an additional clinical trial — to seek approval for ataluren (formerly PTC124) as a treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) caused by non

DMD/BMD — Tom Thompson, Ph.D.

Tom Thompson, associate professor in the department of molecular genetics, biochemistry and microbiology at the University of Cincinnati in Ohio, was awarded an MDA research grant totaling $330,000 over three years. The funds will help support Thompson's study of potential therapies based on blocking a protein called myostatin for Duchenne (DMD) and Becker (BMD) muscular dystrophies.

DMD/BMD — Rita Perlingeiro, Ph.D.

MDA awarded a research grant totaling $390,000 over three years to Rita Perlingeiro, associate professor in the Department of Medicine at the University of Minnesota in Minneapolis. The funds will help support Perlingeiro's study of genetic correction and muscle regeneration in Duchenne (DMD) and Becker (BMD) muscular dystrophies.

DMD/BMD — Joseph Metzger, Ph.D.

MDA awarded a research grant totaling $237,868 over two years to Joseph Metzger, professor and chair of integrative biology and physiology at the University of Minnesota in Minneapolis. The funds will help support Metzger's work in development and testing of copolymer molecules, or "membrane sealants," for Duchenne (DMD), Becker (BMD) and other muscular dystrophies.

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