Neuromuscular Junction Diseases

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?

This second of a series of three stories covering the 2014 MDA Clinical Conference discusses pain in neuromuscular disorders

posted on April 4, 2014 - 9:52am
The 2014 MDA Clinical Conference, held in Chicago March 16-19, was attended by some 500 people, mostly physicians and other health care professionals.

How MDA-supported research to counteract a complement protein and rev up regulatory T cells may improve MG treatment

posted on April 1, 2014 - 9:27am
Quest Spring 2014
"I’m on CellCept, prednisone, Mestinon and IVIG every three weeks," says 38-year-old Rachel Pegram. "Prednisone, which I have taken for more than 22 years now, has very rough side effects. It has caused weight gain, diabetes and glaucoma. I cannot say I have ever gone into remission without drugs, but I believe I have been in a drug-induced remission. I have spent a lot of time in hospitals for...

The National Institutes of Health is conducting a study to develop a questionnaire about motor function in young children who have a neuromuscular disorder

posted on December 2, 2013 - 2:15pm
Update (Jan.
posted on October 3, 2013 - 9:15am
Quest Vol. 20, No. 4
In its Summer 2013 round of grants, MDA awarded 31 new grants totaling $8.5 million. 

Jacobus Pharmaceutical has opened its original, 30-person, invitation-only trial of 3,4-diaminopyridine for Lambert-Eaton myasthenic syndrome (LEMS) to additional participants who meet study criteria

posted on October 1, 2013 - 5:00am
Update (April 14, 2014): This phase 2 trial of 3,4-diaminopyridine is now closed to new participants. However, the drug remains available on a "compassionate use" -- also called "expanded access" -- basis to qualified patients with LEMS and related disorders of the neuromuscular junction. See Treatment Use of 3,4-Diaminopyridine; or enter NCT01765140 in the search box at ClinicalTrials.gov.

Research progress in one disease can lead to progress in other diseases — and MDA’s latest round of grants seek to leverage this potential

posted on August 21, 2013 - 1:35pm
In its summer 2013 round of research grant awards, the Muscular Dystrophy Association aims to catalyze research progress in a dozen neuromuscular diseases, with an eye toward applying that knowledge to related muscle diseases, as well. “A large number of our grants are investigating new therapeutic technologies,” notes Jane Larkindale, MDA's vice president of research. “These are 'platform'...

In the 1970s, Elyse Bruce saw her teenage brother deal with MG — but in the 2000s, she struggled to find a diagnosis for her young son’s mysterious symptoms

posted on June 25, 2013 - 11:42am
I was a pre-teen in the 1970s when my older brother began having problems. My parents thought he was just hitting those teen years when rebellion is the word of the day. He went from being an excellent clarinetist to a mediocre one, and the more he practiced, the worse he sounded. He would get up from the supper table and fall into the wall; my parents asked him straight out if he was on drugs.