April 21, 2011
The state of the science in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease), current lines of research, efforts to establish a TDP43 research mouse colony, and a primer on protein-based
April 20, 2011
Below are brief reports and links to more information about neuromuscular disease research presented at the 63rd annual meeting of the American Academy of Neurology (AAN), held in Honolulu April 9-16
April 15, 2011
Neuralstem's trial of spinal cord injections of stem cells is on track
The first nine participants in a phase 1 (safety and feasibility) trial of neural stem cell transplantation in amyotrophic
April 13, 2011
Most people affected by neuromuscular disease know all too well that their condition is costly. MDA wants to find out just how costly — and then translate those figures into terms that will speak
March 31, 2011
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
March 30, 2011
Update 8/31/11: Enrollment screening for this study has closed to new prospective participants.
"EMPOWER," a large-scale, global phase 3 clinical trial of a molecule called dexpramipexole in ALS (
March 29, 2011
sClinical trials that use compounds called antisense oligonucleotides to cause skipping of exon 51 of the dystrophin gene in individuals with Duchenne muscular dystrophy (DMD) are moving forward in
March 24, 2011
MDA is funding research into a potential treatment for amyotrophic lateral sclerosis (ALS) that inhibits excessive glutamate signaling in the brain and spinal cord.
The grant of $268,000 to Larkspur
March 24, 2011
Three small molecules that normalize muscle structure and function and improve survival in dystrophin-deficient zebrafish have been identified by scientists at Children's Hospital Boston, Harvard
March 23, 2011
TDP43 has unexpectedly extensive role
Scientists have found that the protein TDP43 normally influences the synthesis of more than 1,500 other proteins, including itself. Mutations in the gene for
March 21, 2011
UPDATE (April 9, 2012): MDA and ALS Biopharma agreed to terminate this project in April 2012, when ALS Biopharma determined that it was unable to manufacture its HSP70-based compound in a pure enough
March 18, 2011
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
March 15, 2011
A team of research scientists has found that mice with a disease resembling a severe form of spinal muscular atrophy (SMA) that were treated with a gene-modifying molecule produced more of a needed
March 14, 2011
MDA’s translational research program has announced it is funding research into a potential treatment for the autoimmune disorder myasthenia gravis (MG).
The grant of $530,480 to University of
March 09, 2011
A team of researchers at the David Geffen School of Medicine at the University of California-Los Angeles (UCLA) has demonstrated that the naturally occurring protein interleukin 10 (IL10) may help
March 07, 2011
New research suggests that the DNA of a normally dormant virus may be playing a role in causing amyotrophic lateral sclerosis (ALS).
If the findings are confirmed, they could ultimately have
March 03, 2011
Loss of TDP43's normal function could be ALS contributor
New mouse studies suggest that loss of the normal function of the TDP43 protein, and not just an abnormal "toxic gain of function" of this
March 02, 2011
MDA has awarded $2 million to the ALS Therapy Development Institute (ALS TDI) in Cambridge, Mass., to help scientists complete the preclinical testing of four promising new compounds in ALS TDI's
February 25, 2011
Congenital myasthenic syndromes
A multinational team of scientists has identified mutations in the gene for glutamine-fructose-6-phosphate transaminase 1 (GFPT1) as responsible for some forms of a
February 10, 2011
Charcot-Marie-Tooth disease
Researchers supported in part by MDA have developed flow charts to guide genetic testing for the various subtypes of Charcot-Marie-Tooth disease (CMT), streamlining the
February 10, 2011
New space for research, new mouse models of ALS and new technology to screen therapeutic agents all will play a role in 2011 at the ALS Therapy Development Institute (ALS TDI), said Steve Perrin, ALS
February 09, 2011
A new MDA translational research grant for $476,465 over three years will allow Carmen Bertoni at the University of California, Los Angeles (UCLA) to develop RTC13, an experimental compound designed
February 04, 2011
MDA has awarded eight new grants totaling nearly $2.5 million to fund research projects focused on uncovering the causes of, and developing therapies for, ALS. The effective start date for the grants
February 04, 2011
The Muscular Dystrophy Association has awarded 44 grants totaling $13.5 million to support research efforts aimed at advancing understanding of disease processes and uncovering new strategies for
February 02, 2011
Isis Pharmaceuticals hosted an interactive webinar Wednesday, March 2, 2011, at 12 noon Eastern Standard Time, to help prospective participants understand its MDA-supported, phase 1 study of ISIS-