Duchenne Muscular Dystrophy (DMD)

DMD: What's Next for Drisapersen?

Eteplirsen Data Still Strong: Treated Boys with DMD Stable at 120 Weeks

DMD: Prosensa to Present Webcasts on Drug Development

Dutch biopharmaceutical company Prosensa, developer of the experimental drug drisapersen and other experimental compounds for Duchenne muscular dystrophy (DMD), will update the investor community via a Thursday, Jan. 16, 2014, presentation at the J.P. Morgan Healthcare Conference in San Francisco. The presentation will be webcast at 10 a.m. Pacific time/1 p.m. Eastern time.

On Tuesday, Jan. 21, at 8 a.m. Eastern time, Prosensa will conduct a separate webinar for DMD patients and families.

DMD: GSK Returns Drisapersen Development Rights to Prosensa

The rights to development of the experimental Duchenne muscular dystrophy (DMD) drisapersen have now been returned to the drug's original developer, Dutch biotechnology company Prosensa. The rights were formerly jointly held by Prosensa and multinational pharmaceutical company GlaxoSmithKline (GSK).

College Course on DMD Available Online

A course on Duchenne muscular dystrophy (DMD), geared toward graduate and upper-division undergraduate students and held at Ohio State University College of Medicine and Nationwide Children's Hospital in Columbus, is open to the public, without charge, via real-time streaming and podcasts.

HT-100 Clinical Program Status Changed

Trial of Tadalafil in DMD Open to Participants

DMD/BMD: Stop Codon Read-Through Drug Trial Still Recruiting Participants

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.

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