A newly developed research mouse that has the same combination of genetic alterations that causes human facioscapulohumeral muscular dystrophy (FSHD) is expected to change the way research in this disease is conducted, possibly speeding the development of therapies.
An online survey about ALS symptoms, now open to people with amyotrophic lateral sclerosis (ALS), is being conducted by the Neurological Clinical Research Institute at Massachusetts General Hospital (MGH) in Boston.
Scientists continue to work at uncovering the biological mechanisms underlying amyotrophic lateral sclerosis (ALS). One area of intense study involves the potential role of central nervous system support cells called astrocytes.
The news is generally favorable for many important government-funded neuromuscular disease research projects, in the wake of Congress’ recent passage of a continuing resolution funding the government through the end of September.
The Muscular Dystrophy Association and The ALS Association (ALSA) submitted a joint comment to the U.S. Food and Drug Administration (FDA) today, commending and thanking the agency for convening its first-ever ALS public hearing on Feb.
The latest advances in brain and central nervous system research were discussed at the 65th annual meeting of the American Academy of Neurology, held in San Diego, March 16-23, 2013.
An experimental drug designed to treat the underlying molecular defect in spinal muscular atrophy (SMA) has shown encouraging results in a phase 1 trial.
A therapeutic strategy that combines gene therapy and stem cell transplantation has shown encouraging results in mice with a disorder mimicking Duchenne muscular dystrophy (DMD).
R. Rodney Howell — respected geneticist, pediatrician and chairman of the MDA Board of Directors — has been honored by the March of Dimes for playing a key role in developing the uniform panel of serious disorders for which nearly every baby in the United States now is tested.
Proposals exploring the feasibility and advisability of implementating newborn screening for two disorders in MDA's program — Duchenne muscular dystrophy and
Spinal muscular atrophy (SMA) is a disease in which nerve cells that control muscles (motor neurons) in the spinal cord die, causing progressive weakness in the voluntary muscles.
An international patientregistry (database) is gathering information about children and adults with any form of congenital muscular dystrophy (CMD), a congenital my