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.
A drug that has FDA approval for other indications is being tested in CCD patients who are at least 7 years old
The National Institute of Nursing Research (NINR) at the National Institutes of Health in Bethesda, Md., is conducting a study to determine if an antioxidant drug that has been approved by the U.S. Food and Drug Administration for other indications can reduce the severity of some symptoms in people with central core disease (CCD), a genetic disease caused by defects in a structure known as...