Can toxic genes be blocked to treat disease?
posted on January 1, 2007 - 3:02pm
Since the 1990s, gene therapy - the insertion of functional genes to compensate for nonfunctional ones - has been the goal of researchers working in several muscular dystrophies, spinal muscular atrophy, Friedreich's ataxia, metabolic muscle diseases and myotubular myopathy.
High levels of misplaced proteins may worsen the disease process in myasthenia gravis
posted on August 25, 2009 - 12:22pm
Stepped-up production of fragments of acetylcholine receptors, the microscopic "landing pads" on muscle fibers that normally help process signals from the nervous system, may provoke the immune system and worsen myasthenia gravis (MG), new research shows.
In mice with an MG-like disease, these receptor fragments (which are proteins) are abundant but nonfunctional, and they end up in the wrong...
Once incorrectly diagnosed and improperly treated, they're now among the most treatable neuromuscular disorders
posted on June 1, 2001 - 12:05pm
When Phillip Martin was a baby, most people — even doctors — didn't notice anything unusual about him, except perhaps that he was a little quiet compared to other babies. But as they watched him grow, Richard and Daina Martin of Kelseyville, Calif., began to sense their son might have a physical disability.
Phillip couldn't hold his head up by himself until he was nearly 6 months old, Daina...
High levels of misplaced proteins may worsen the disease process in MG.
posted on August 20, 2009 - 5:00pm
Stepped-up production of fragments of acetylcholine receptors, the microscopic "landing pads" on muscle fibers that normally help process signals from the nervous system, may provoke the immune system and worsen myasthenia gravis (MG), new research shows.
In mice with an MG-like disease, these receptor fragments (which are proteins) are abundant but nonfunctional, and they end up in the wrong...
Thirty-eight new grants support research in ALS, central core disease, spinal muscular atrophy, muscular dystrophy, and other diseases in MDA's program
posted on August 17, 2010 - 6:39pm
MDA has awarded 38 new research grants totaling more than $14 million and covering more than a dozen neuromuscular diseases.
MDA's Board of Directors met in Los Angeles July 16, where it reviewed and approved the new grants based on recommendations from the MDA Scientific and Medical Advisory Committees. Grants were scored and recommended for approval based on the capabilities of the...
News on Duchenne muscular dystrophy, Friedreich's ataxia, autoimmune diseases, and cell and gene therapies
posted on September 24, 2010 - 1:39pm
Duchenne muscular dystrophy
News about research in Charcot-Marie-Tooth disease, congenital myasthenic syndromes, Duchenne/Becker MDs, Friedreich's ataxia, Pompe disease and spinal-bulbar muscular atrophy
posted on February 10, 2011 - 11:09am
Charcot-Marie-Tooth disease
An MDA-supported open-label trial found the asthma drug benefited 17 of 18 people with either of two forms of congenital myasthenic syndrome
posted on November 9, 2011 - 2:56pm
Researchers have found that the drug albuterol appears to be beneficial in two forms of congenital myasthenic syndrome (CMS) — CMS related to mutations in the collagen Q (colQ) gene and CMS related to mutations in the DOK7 gene.
In this first of several reports on MDA's Clinical Conference, the role of genetics and immunology in different neuromuscular diseases is described
posted on March 19, 2012 - 3:08pm
More than 500 physicians, allied health care professionals and MDA staff attended the MDA's 2012 Clinical Conference in Las Vegas, March 4-7.
The program emphasized:
Encouraging findings about eculizumab and GM-CSF were presented
posted on May 2, 2012 - 11:49am
The 2012 annual meeting of the American Academy of Neurology, held in New Orleans April 21-28, included findings related to myasthenia gravis (MG).
