[title]
SMA: Participants Sought for Study About Recreational Activities
October 25, 2012
Investigators at Northwestern University in Chicago are seeking people with spinal muscular atrophy (SMA), ages 2 to 21, to participate in a study about preferences and involvement in recreational and leisure activities.  
[title]
'Guided Missile' Strategy for MG Shows Promise in Mice
October 25, 2012
Researchers funded in part by MDA say a gene-based therapy designed to treat myasthenia gravis (MG) has shown promise in mice with an MG-like disease.
[title]
Anti-Cancer Drugs May Help Build Muscle
October 18, 2012
[title]
ALS Researcher Elected to Institute of Medicine
October 18, 2012
[title]
New York Post Awards Top Honor to National Disability Leader
October 17, 2012
[title]
Dysfunction of Motor Circuits May Underlie SMA
October 17, 2012
[title]
Ventricular Assist Device Implanted in Man with Duchenne MD
October 16, 2012
[title]
Nationwide Children’s Podcast Explores the Congenital Muscular Dystrophies
October 15, 2012
 
[title]
Interim Results Suggest Chaperone Drug Enhances Enzyme Treatment in Pompe Disease
October 12, 2012
 
[title]
Neural Stem Cell Trial in ALS ‘Very Successful’ So Far
October 12, 2012
Results from a completed phase 1 trial of neural stem cells in people with amyotrophic lateral sclerosis (ALS) show that the stem cells and the surgical method used to transplant them were safe and well-tolerated.
[title]
Press Release: Drug for Duchenne Muscular Dystrophy Successfully Completes Phase 1 Trial; Trial in Boys with DMD May Be Next
October 10, 2012
[title]
DMD: Reformulated Utrophin Upregulator Looks Good in Healthy Volunteers
October 10, 2012
[title]
DMD Imaging Study Open at Three Sites
October 08, 2012
A multicenter study of the use of magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) to measure the progression of Duchenne muscular dystrophy (DMD) is open at sites in Florida, Oregon and Pennsylvania for boys with DMD who meet study criteria.
[title]
CMD, LGMD: 'Conditional Knockout' Mouse Will Help Researchers Study Fukutin Deficiency
October 08, 2012
By disrupting the fukutin gene at different time points in mice embryo, researchers have been able to develop research models of two types of human muscle disease: Fukuyama congenital muscular dystrophy (Fukuyama CMD)  and
[title]
Americans Put Their Strength Behind MDA to Fight Muscle Diseases
October 05, 2012
[title]
Grants for In-Home Care Available from Philanthropist with LGMD
October 05, 2012
A small nonprofit organization is offering limited grants for in-home care to people with physical disabilities. The next quality-of-life grant application deadline is November 1, 2012.
[title]
Sizing Up Early Enzyme Replacement for Pompe Disease
October 04, 2012
A study of 11 children with infantile-onset Pompe disease (acid maltase deficiency) who started enzyme replacement therapy by the time they were 6 months old has shown the treatment can markedly improve the course of the disease, but that residual
[title]
ALS, SMA Share a Biochemical Pathway
October 04, 2012
New evidence links the motor neuron diseases ALS (amyotrophic lateral sclerosis) and spinal muscular atrophy (SMA), an international team of scientists has reported.
[title]
DMD: Eteplirsen Results Very Encouraging at 48 Weeks
October 03, 2012
The biopharmaceutical company Sarepta Therapeutics announced today that its experimental exon-skipping compound, eteplirsen, resulted in an increase in dystrophin and  "significant clinical benefit" on the six-minute walk test in a phase 2b trial in boys with Duche
[title]
A Closer Look at the 48-Week Eteplirsen Trial Results
October 03, 2012
The experimental exon-skipping drug eteplirsen, in development by Sarepta Therapeutics to treat approximately 13 percent of boys with Duchenne muscular dystrophy (DMD), has been found to have significant functional and biochemi
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