How MDA-supported research to counteract a complement protein and rev up regulatory T cells may improve MG treatment
"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
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
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
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
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.
Carmelita Pallares, 31, of Albuquerque, New Mexico, finds ways to conserve precious energy so she can care for her toddler daughter
My name is Carmelita Pallares. I am Native American; I am a member of both the Navajo Nation and Santo Domingo Pueblo tribe. I am 31 years old. I was diagnosed seven years ago with myasthenia gravis, only three months after having emergency surgery and undergoing a temporary colostomy following an accident. My neurologist believed that the trauma my body suffered from this accident and the stress...