A look at symptoms, diagnosis, treatments and current MDA-supported research into this complex autoimmune disorder

posted on June 15, 2015 - 1:01pm
In recognition of June being National Myasthenia Gravis Awareness Month, MDA is examining the disease from several perspectives. This article offers an overview of signs, symptoms, diagnosis and treatments for myasthenia gravis (MG), as well as information on drugs that people with MG should avoid.

Yearlong, MDA-supported study comparing two prednisone dosing schedules in Duchenne MD found similar benefits and side effects

posted on July 20, 2011 - 9:52am
A one-year, MDA-supported study comparing a weekend-only prednisone treatment schedule with a daily prednisone schedule in boys with Duchenne muscular dystrophy (DMD) has found that the two treatment regimens provide about the same benefits and have approximately the same side-effect profile.

Investigators are testing compounds designed to cause exon skipping in Duchenne MD and drugs that may increase blood flow in Duchenne and Becker MD

posted on June 21, 2011 - 9:08am
U.S. trial of eteplirsen for Duchenne MD delayed

Physicians and scientists will meet Jan. 21-22, 2011, in Columbus, Ohio, at an MDA-sponsored conference about cardiomyopathy in Duchenne muscular dystrophy

posted on January 18, 2011 - 4:06pm
Experts from around the world will gather Jan. 21-22, 2011, at an MDA-sponsored conference about the heart in Duchenne muscular dystrophy (DMD).
posted on January 1, 2011 - 12:47pm
QUEST Vol. 18, No. 1
R. Mark Payne, M.D., professor of pediatrics at Riley Heart Research Center, Indiana University, is a pediatric cardiologist whose medical specialty is intervention in children with heart disease, and who conducts research on heart disease due to mitochondrial defects. In Friedreich’s ataxia (FA), heart problems are related to a deficiency of the frataxin protein, which is normally located in...
posted on January 1, 2011 - 12:33pm
QUEST Vol. 18, No. 1
Overall, idebenone has been shown to be safe and well-tolerated in a number of phase 1, 2 and 3 human clinical trials dating back to 1999. The most common side effects are mild to moderate gastrointestinal issues such as diarrhea, nausea and indigestion. Early trials at low doses first demonstrated the potential for idebenone to reduce FA-associated cardiomyopathy (heart muscle abnormality).
posted on October 1, 2010 - 12:25pm
QUEST Vol. 17, No. 4
Featured in this issue: First U.S. exon-skipping trial opens * Utah researchers seek families with Ullrich CMD or Bethlem myopathy * Acceleron receives Fast Track, Orphan Drug designations for ACE031 * BioMarin abandons BMN195 development
posted on June 30, 2010 - 4:41pm
QUEST Vol. 17, No. 3
If you are pregnant or plan to become pregnant, discuss with your physician any medications you are taking. In all cases where medication is required, the risk to the fetus must be weighed against the health of the mother.