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.
Boys with Duchenne muscular dystrophy (DMD) participating in a phase 2b extension trial of the experimental exon-skipping drug eteplirsen showed continued stabilization of walking distance at 120 weeks.
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.
"We are undoubtedly slowing down the MND [motor neuron disease] supertanker, and can start to see how we might turn it around for the first tim," said Martin Turner, senior clinician scientist at the University of Oxford (United Kingdom), on research being reported on at the 24th International
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.
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.