Limb-Girdle Muscular Dystrophy (LGMD)

Race, Cardiomyopathy Shorten Life Span in MD

A new study reported by the Centers for Disease Control and Prevention (CDC) shows that survival time has significantly increased for certain categories of people with muscular dystrophy (MD) but that race and cardiac status have a large impact on survival.

Study Seeks People With Uncertain MD Diagnoses

A study to determine the early features of late-onset Pompe disease (acid maltase deficiency) is seeking 250 adults who have a clinical diagnosis of unclassified limb-girdle muscular dystrophy (LGMD), an uncertain diagnosis of other forms of muscular dystrophy (MD),or an unclassified myopathy(muscle disease)who do not carry any biochemical, metabolic, enzymatic, serologic (blood), molecular or pathologic diagnostic marker that confirms their diagnosis.

MDA Awards More Than $14 Million in Research Grants

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 applicant, the scientific merit of the project, and the proposal's relevance to developing treatments for the disease. The effective start date for all grants was July 1, 2010.

Biker with LGMD Creates His Own Accessible Motorcycle

The “passenger” in Steve Williams’ motorcycle sidecar doesn’t say much, but if it could talk, it probably would have plenty of stories to tell.

 Steve Williams explains how he and his brother outfitted this Honda Nighthawk with a special wheelchair-carrying sidecar.

The passenger is Williams’ manual wheelchair.

DMD Research: New Funding for Imaging Studies

The use of magnetic resonance imaging (MRI) as an assessment tool in boys with Duchenne muscular dystrophy (DMD) is being studied by former MDA grantee Krista Vandenborne, through a $7.5 million grant from the National Institutes of Health (NIH).

The goal of the study is to assess whether MRI technology can be used as a precise, noninvasive measure of muscle tissue, gauging both disease progression and the effectiveness of therapies tested in children with DMD.

A Beach Park for EveryBODY

Designers of a new accessible beach park in Virginia Beach, Va., are very glad they asked Caroline Pennell, of Chesterfield, to look over their plans. The 14-year-old with limb girdle muscular dystrophy (LGMD) came up with the best idea of all. 

Gentamicin Trial in DMD Shows Mixed Results

An MDA-supported clinical trial of intravenous gentamicin in children and adolescents with a form of Duchenne muscular dystrophy (DMD) caused by so-called "nonsense" mutations (also called "premature stop codon" mutations) found the drug was safe and that levels of the muscle protein dystrophin increased in some, but not all, participants who received the drug for six months.

MDA Awards $21 Million for Research

In December 2009, MDA awarded $21 million in new research grants for neuromuscular disease research.

MDA's Scientific Advisory Committee (SAC) and Medical Advisory Committee (MAC) meet each fall and spring to review applications for research grants. Applications are scored on the basis of the capabilities of the applicant, the scientific merit of the project, and the proposal's relevance to developing treatments for the diseases in MDA's program. MDA's Board of Directors then reviews the recommendations of the MAC and SAC.

New Grant For LGMD2D Gene Therapy

Development of delivery of a therapeutic gene via the bloodstream to the thigh muscles in people with type 2D limb-girdle muscular dystrophy (LGMD2D) is proceeding on schedule, thanks in part to a new $458,814 grant from MDA to neurologist Jerry Mendell at Nationwide Children's Hospital in Columbus, Ohio.

MDA's Board of Directors approved the new funding, via the Association's translational research/MDA Venture Philanthropy program, on Dec. 4, 2009.

Gene Modifies Severity of LGMD2C

New research has shown that a change in a gene not previously connected to type 2C limb-girdle muscular dystrophy (LGMD2C) modifies the severity of the disease in mice and is likely to do the same in people with this and perhaps with related types of muscular dystrophy.

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