MDA-supported researchers have developed a mouse with a mutation in a muscle sodium channel and a disease that mimics human hypokalemic PP
posted on October 14, 2011 - 10:15am
MDA grantee Stephen Cannon at the University of Texas Southwestern Medical Center in Dallas coordinated a study team that has developed a mouse model of one type of hypokalemic periodic paralysis, publishing the development in the Oct. 3, 2011, issue of the Journal of Clinical Investigation.
A phase 3 trial of the drug dichlorphenamide in hyperkalemic and hypokalemic periodic paralysis is open at several U.S. and European centers
posted on October 14, 2011 - 10:13am
A multicenter study of the drug dichlorphenamide in 140 adults with hyperkalemic or hypokalemic periodic paralysis is open at sites in California, Kansas, Massachusetts, Minnesota, Missouri, New York, Ohio and Texas, as well as in France (not yet recruiting), Italy and the United Kingdom.
The conference, Nov. 4-6 in Orlando, Fla., will update families on research and address daily life issues
posted on August 5, 2011 - 3:33pm
The 2011 Periodic Paralysis Association Conference will be held Nov. 4-6 in Orlando, Fla., and is geared toward people with periodic paralysis (PP), their families, physicians and friends.
This is not an MDA meeting, but several current and former MDA research grantees will be speaking. Among them are:
Quest takes a look at gene therapy, 'antisense' and other cutting-edge scientific approaches and how they're being applied to diseases in MDA's program
posted on July 1, 2011 - 4:15pm
Antisense oligonucleotides block flawed genetic instructions
Antisense oligonucleotides — also called antisense, oligos, or simply AONs — are pieces of genetic code that keep other genetic code from being processed. Designed to pair up with a particular sequence of DNA or RNA, AONs can change, block or destroy targeted genetic instructions in a variety of ways.
Wake Forest researcher receives $369,365 for preclinical testing of gene transfer therapy for myotubular myopathy
posted on May 26, 2011 - 2:16pm
MDA’s translational research program has announced it is funding research into a potential treatment for the inherited muscle disorder X-linked myotubular myopathy (MTM).
The grant of $369,365 grant to Wake Forest University professor Martin Childers will fund a three-year study of myotubularin gene transfer in mice and dogs that have an MTM-like disease.
News briefs about research in Charcot-Marie-Tooth disease, hereditary inclusion-body myositis, limb-girdle muscular dystrophy, myotubular/centronuclear myopathies and Pompe disease
posted on March 31, 2011 - 10:20am
Charcot-Marie-Tooth disease
A two-year, large-scale trial of ascorbic acid (vitamin C) in people with type 1A Charcot-Marie-Tooth disease (CMT1A) conducted in Italy and the United Kingdom has found the substance had no significant effect on the disease compared with a placebo. Ascorbic acid was taken orally at 1.5 grams per day in this study. An ongoing U.S.-based trial (now closed to recruitment...
An MDA-sponsored meeting explored progress in five key therapeutic strategies under development for neuromuscular diseases
posted on March 18, 2011 - 3:30pm
Moving therapeutic strategies from the laboratory to clinical trials and ultimately to the market as treatments was the theme of the MDA National Scientific Conference held March 13-16, 2011, in Las Vegas.
Some 300 people attended the conference, the first in a planned series of such MDA-sponsored meetings that will emphasize new research and current medical care. The majority of presenters and...
