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.
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
The Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) is actively seeking people with amyotrophic lateral sclerosis (ALS) to participate in a phase 2 clinical trial designed to evaluate whether
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 po
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
Results from three recent studies — two supported in part by MDA — shed light on how the chromosome 9 open reading frame 72 (C9ORF72) mutation causes nerve cell death in amyotrophic lateral sclerosis (ALS) and
Summit PLC, a biotechnology company based in Abingdon, United Kingdom, has announced it has received approval from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) and Ethics Review Committee to begin testing its experimental drug SMT C1100 in children with
Trial investigators are actively seeking people with amyotrophic lateral sclerosis (ALS) to participate in a phase 2 clinical trial that will evaluate the effects of a five-drug treatment regimen used to suppress immune system activity.