Update (June 1, 2012)— The brief Drugs help DMD-related cardiomyopathy has been updated to include a May 2012 podcast provided by Nationwide Children's Hospital's "This Month in Muscular Dystrophy."
Update (June 21, 2012):This story was updated to include the fact that MDA funding helped support the phase 1 trial, as well as the preclinical development of RG3039.
Top scientists and clinicians from around the world are discussing the latest research in neuroscience, and the care of individuals with nerve and muscle diseases, at the 2012 annual meeting of the American Academy of Neurology in New Orleans, April 21-28.
Update (May 25, 2012) —Summit announced today that the first group of healthy volunteers in the phase 1 trial of its newly formulated SMT C1100 has begun receiving the drug. The dose-escalating trial will evaluate whether the new formulation of SMT C1100 is safe and whether consistent blood levels of the drug can be achieved.Results are expected by the end of the year.
Several experts presented their views of "best practices" for care of people with neuromuscular disorders at MDA's 2012 Clinical Conference, held in Las Vegas March 4-7.
Many questions remain about optimal care in these disorders, but it's clear that attention to heart and respiratory function are of paramount importance.
It’s about the children, their health and their futures.
That’s long been the philosophy of R. Rodney Howell, M.D., renowned pediatrician and geneticist, and current chairman of the Board of Directors for the Muscular Dystrophy Association.
SOD1 causes toxicity in some familial and some sporadic ALS
In some people with bulbar-onset sporadic ALS, changes to the SOD1 protein cause the same toxicity as is generated by mutated SOD1 protein in the familial (inherited) form of the disease, a team of researchers has reported.