A look at symptoms, diagnosis, treatments and current MDA-supported research into this complex autoimmune disorder
In recognition of June being National Myasthenia Gravis Awareness Month, MDA is examining the disease from several perspectives. This article offers an overview of signs, symptoms, diagnosis and treatments for myasthenia gravis (MG), as well as information on drugs that people with MG should avoid.
A committee convened by the American Academy of Neurology has published new recommendations for doctors who see children with congenital muscular dystrophy
This spring, the American Academy of Neurology (AAN) released a guideline for the diagnosis and care of children with congenital muscular dystrophy (CMD), a group of early-onset, genetic disorders that can involve skeletal (including respiratory) muscle weakness, heart problems, brain and eye abnormalities, and contractures (frozen joints).
An early-onset disease with many genetic causes
Santhera Pharmaceuticals is testing anti-cell death compound omigapil in children and teens with merosin-deficient and Ullrich congenital muscular dystrophies
Santhera Pharmaceuticals continues to recruit participants for a phase 1 trial of omigapil in children and adolescents with the type 1A form of congenital muscular dystrophy (CMD)
Israel-based BioBlast Pharma says a California site will be added soon to locations already testing Cabaletta in oculopharyngeal muscular dystrophy in Israel and Canada
BioBlast Pharma, based in Tel Aviv, Israel, has been given clearance from the U.S. Food and Drug Administration (FDA) to test its experimental drug Cabaletta in the U.S. in people with oculopharyngeal muscular dystrophy (OPMD).
The experimental antisense drug will be tested in infants with a genetic spinal muscular atrophy diagnosis who are to date without symptoms
Biogen Idec has announced plans to run a phase 2 clinical study of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA)who are less than 6 weeks old and have a genetic diagnosis of the disorder but are not yet showing symptoms.
MDA-supported researcher Charles Gersbach and colleagues have developed a strategy to change dystrophin DNA that has shown promise in the lab
MDA research grantee Charles Gersbach, assistant professor of biomedical engineering at Duke University, and colleagues, recently announced an advance in gene modification that could turn out to be a game-changer for boys and young men with Duchenne muscular dystrophy (DMD). The team's results were published Feb.