Health Care

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.

Many MDA grants for research in myasthenia gravis aim to prevent the immune system from attacking needed muscle proteins while allowing it to fight infection

posted on June 25, 2013 - 11:00am
It can start with nothing more than a drooping eyelid or a slight slurring of speech, either of which can come and go and improve with rest. But it can progress, often gradually over weeks or months, to affect all the voluntary muscles, including those controlling breathing. Weakness may be minimal early in the morning after a good night's sleep and worsen throughout the day, especially after...

Researchers look at the 'window of opportunity' for effective treatment in spinal muscular atrophy and the therapeutic potential of celecoxib

posted on June 20, 2013 - 4:27pm
Recent spinal muscular atrophy (SMA) research includes findings that shed additional light on the optimal "window of opportunity" for treatment and point toward a potential candidate for therapeutic development.

Disease registries are seeking participants with CMD, FSHD, LGMD, MMD and Pompe disease

posted on June 7, 2013 - 11:05am
FSHD, MMD: Registry remains open The University of Rochester Medical Center in New York state would like to remind people with facioscapulohumeral muscular dystrophy (FSHD) or myotonic muscular dystrophy (MMD, or DM), and their families, about its registry to advance research in these two disorders.

Nighttime respiratory problems may be more common in people with Friedreich's ataxia than in the general population, necessitating the need for regular screening

posted on June 4, 2013 - 11:49am
Results from a study conducted in Australia show that a nighttime respiratory problem called obstructive sleep apnea occurs more frequently in people with Friedreich's ataxia (FA) than in the general population.

Results from a 20-person pilot trial suggest that clenbuterol may improve motor and respiratory function in people with spinal-bulbar muscular atrophy, but safety concerns were raised

posted on May 29, 2013 - 4:27pm
A 20-person, open-label pilot trial conducted in Italy and designed to test the safety, tolerability and efficacy of clenbuterol in people with spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) found that the drug was well-tolerated, although some possible safety issues were identified. Trial investigators note that the study findings suggest a possible “positive effect on SBMA disease...

Federal committee votes to add the enzyme deficiency disorder to the list of recommended conditions for which states screen newborns

posted on May 17, 2013 - 2:45pm
The Discretionary Advisory Committee on Heritable Disorders in Newborns and Children (DACHDNC) today voted to add Pompe disease (acid maltase deficiency) to a list of diseases that it recommends states screen for in newborns.

The AAP and the ACMG have issued recommendations on updated technologies and new uses of genetic testing and screening

posted on March 6, 2013 - 2:15pm
As scientists learn more about what our DNA can tell us about health and disease, public interest has intensified and genetic testing has become increasingly common. In response, the American Academy of Pediatrics (AAP) and the American College of Medical Genetics and Genomics (ACMG) have released new guidelines to address updated technologies and new uses of genetic testing and screening in...