Duchenne Muscular Dystrophy (DMD)

Blood-Filtering Procedure Removes Antibodies That Impede Gene Transfer

A research group supported in part by MDA has found that a blood-filtering strategy known as plasmapheresis looks promising as a way to help overcome one type of unwanted immune response to gene transfer therapy. Gene transfer therapy is the addition of therapeutic genes to treat disease, and it is often administered via transport vehicles made from viruses.

Louis Chicoine at Nationwide Children's Hospital and Ohio State University in Columbus, Ohio, and colleagues, reported the findings online Oct. 23, 2013, in the journal Molecular Therapy.

Study Seeks Parents, Guardians of Young Children with Neuromuscular Disease

The National Institute of Nursing Research, part of the National Institutes of Health (NIH) in Bethesda, Md., is seeking parents or guardians of children from birth through age 5 with a neuromuscular disease for a study to develop a questionnaire about motor function in young children.

DMD/BMD: Summit and University of Oxford to Collaborate on Drug Development

MDA-supported biotechnology company Summit PLC has entered into a collaboration with the University of Oxford (United Kingdom) for continued development of utrophin modulators to treat Duchenne muscular dystrophy (DMD) and possibly Becker muscular dystrophy (BMD).

Prosensa Provides Exon-Skipping Program Update to DMD Community

BMD, IBM: Outcome Measures Study Seeks Participants

A study to determine the best outcome measures — ways to evaluate the effects of a treatment — for use in clinical studies in Becker muscular dystrophy (BMD) and sporadic (nongenetic) inclusion-body myositis (sIBM) is underway at Nationwide Children's Hospital in Columbus, Ohio, under the supervision of neurologist Jerry Mendell working with physical therapists Linda Lowes and

Prosensa, GSK Conducting DMD Natural History Study

MDA Funds Development of Drug to Fight Inflammation, Scarring in DMD

Halo Therapeutics Receives $500,000 MDA Award to Develop Drug for Duchenne Muscular Dystrophy

DMD — Halo Therapeutics

MDA awarded a $500,000 grant to biotechnology company Halo Therapeutics for development of a treatment for Duchenne muscular dystrophy (DMD). The two-year grant begins in November 2013 and will run through Nov. 15, 2014. The Newton, Mass., company is developing HT-100, an experimental compound designed to combat excessive inflammation and formation of scar tissue in muscle and promote muscle regeneration.

RE: FDA Says That New Drug Application Filing for Eteplirsen is Premature