posted on April 1, 2009 - 12:26pm
Story includes research items about: Charcot-Marie-Tooth disease, congenital muscular dystrophy, Duchenne muscular dystrophy, Emery-Dreifuss muscular dystrophy (EDMD), type 1 myotonic muscular dystrophy, and spinal muscular atrophy - including, type 3 SMA and spinal-bulbar muscular atrophy (SBMA, Kennedy disease).
posted on September 1, 2007 - 9:47pm
posted on March 1, 2007 - 2:34pm
An MDA-supported research team, testing gene transfer in dogs using adeno-associated viral (AAV) shells (vectors), noted an unwanted immune response, a factor they recommend be considered in human trials. The response occurred even when the vectors weren’t carrying any genes.
Until now, AAV transporters have been thought not to provoke much of an immune response.
posted on July 1, 2010 - 4:03pm
SMA Research: Raising SMN Levels Three Ways
Recent research in spinal muscular atrophy (SMA) has concentrated on three different ways to raise levels of the protein known as SMN that’s required for the health of nerve cells that control muscle function.
Injections of genes for the muscle-growth protein follistatin strengthened leg muscles in monkeys
posted on November 12, 2009 - 3:19pm
Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength with no adverse effects, say researchers at Nationwide Children's Hospital in Columbus, Ohio, and Ohio State University.
The findings could have implications for people with muscular dystrophies and other muscle...
A small-molecule compound counteracts some of the effects of abnormal genetic instructions in MMD1
posted on November 4, 2009 - 3:30pm
A compound that has the potential to be refined and modified into a treatment for type 1 myotonic dystrophy (MMD1, or DM1) has been identified by researchers at the University of Oregon in Eugene, and the University of Rochester (N.Y.) School of Medicine and Dentistry.
posted on October 22, 2009 - 3:47pm
A technique called exon skipping shows great potential to increase muscle strength and prolong life in people with a severe form of Duchenne muscular dystrophy (DMD). According to a study that included MDA-supported Stephen Wilton at the University of Western Australia, exon skipping improves production of a crucial muscle protein that’s missing in people with DMD. For the first time, these...
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...
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
posted on March 31, 2011 - 10:04am
In this issue: MDA awards 44 new research grants * A recently discovered gene variant may indicate severity of DMD * Heart care in Duchenne MD and Becker MD addressed at MDA-sponsored meeting * Duchenne-Becker 'read-through' drug to begin development * Flow charts may aid in diagnosing CMT
