Familial ALS

Stanford University is collecting samples of blood, muscle and other tissues from people with neuromuscular disorders for use in research

posted on January 7, 2015 - 12:06pm
Ever wondered how someone with a neuromuscular disorder in his or her family might contribute to research efforts in this field?

Top researchers are discussing notable developments in neuroscience, including research in many neuromuscular diseases, at the annual American Academy of Neurology meeting April 21-28

posted on April 24, 2012 - 4:00am
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.

Knowing your exact genetic diagnosis does more than just satisfy your curiosity — it can impact family planning, disease management and participation in clinical trials

posted on February 28, 2012 - 2:02pm
Respondents to MDA’s Transitions Survey — in other words, people with a neuromuscular disease who are in their teens through late 30s — made it clear they had questions about the genetics of their disease, as well as questions about family planning and the value of diagnostic testing. MDA’s Genetic Counseling Webinar, which occurred Feb. 22, 2012, answers many of those questions.

Research items about Friedreich's ataxia, myasthenia gravis, mitochondrial myopathies, type 1 myotonic dystrophy, gene therapy and gene modification

posted on July 7, 2011 - 10:28am
Edison drugs target FA, mitochondrial diseases

An MDA-sponsored meeting explored progress in five key therapeutic strategies under development for neuromuscular diseases

posted on March 18, 2011 - 3:30pm
Moving therapeutic strategies from the laboratory to clinical trials and ultimately to the market as treatments was the theme of the MDA National Scientific Conference held March 13-16, 2011, in Las Vegas. Some 300 people attended the conference, the first in a planned series of such MDA-sponsored meetings that will emphasize new research and current medical care. The majority of presenters and...

VCP gene mutations have been linked to some cases of inherited ALS; VCP mutations also are known to cause a form of inclusion-body myopathy 

posted on December 22, 2010 - 9:53am
A multinational study group, using cutting-edge "exome sequencing" technology, has uncovered five mutations in the valosin-containing protein (VCP) gene and implicated them as molecular causes of some familial forms of ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

Scientists know more about familial ALS (FALS) than sporadic ALS; FALS research may have application for both forms of the disease, however. 

posted on April 2, 2010 - 2:21pm
The state of research in the familial (inherited) form of ALS was the focus of a special one-hour Webinar presented by the ALS Therapy Development Institute (ALS TDI) on March 25, with reports by CEO & Chief Scientific Officer Steve Perrin.

ALS TDI investigators have found that disrupting part of the immune system slows disease progression in mice with an ALS-like disease

posted on March 29, 2010 - 2:22am
Blocking a key molecular pathway that the body uses to amplify an immune response has been found to delay disease onset and extend survival in mice with a disease that mimics human amyotrophic lateral sclerosis (ALS), say researchers at the MDA-supported ALS Therapy Development Institute (ALS TDI) in Cambridge, Mass.