PTC Therapeutics' phase 3 trial of its experimental drug ataluren remains open to boys with Duchenne or Becker MD caused by premature stop codon mutations
PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, wants to remind families affected by Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) caused by specific genetic mutations that its phase 3 trial of
An MDA-supported research team has found in animal experiments that plasmapheresis can reduce the number of unwanted antibodies to gene therapy delivery vehicles
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'...
The National Institutes of Health is conducting a study to develop a questionnaire about motor function in young children who have a neuromuscular disorder
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.
Investigators conducting an online survey to gather information from people with inclusion-body myositis report early results and ask a few more questions
Investigators conducting an online survey launched in January 2013 to gather anonymous information from people with inclusion-body myositis (IBM) are now reporting preliminary results and are asking the original respondents to complete a short supplementary survey.
MDA-supported biotechnology company Summit PLC and the University of Oxford will collaborate on development of utrophin modulators to treat Duchenne/Becker MD
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).
Researchers supported in part by MDA have identified antibodies to the LRP4 protein as a third known cause of myasthenia gravis
A research team supported in part by MDA has shown that an abnormal reaction of the immune system against a protein called LRP4 can be added to the known causes of myasthenia gravis (MG), a disorder involving fluctuating weakness and fatigue because of impaired nerve-to-muscle communication. MG is an autoimmune disease, meaning it's caused by abnormal activity of the body's immune system against...