Duchenne Muscular Dystrophy (DMD)

DMD, BMD: Trial of Ataluren in Nonwalkers

Update (March 3, 2010): An update to the article Ataluren Results Disappointing was posted.

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.

Exon-Skipping Drug Delivers Again

Interim results from a human clinical trial of the exon-skipping compound AVI4658 in boys with Duchenne muscular dystrophy (DMD) show that when the compound is delivered to the whole body via the bloodstream — rather than simply injected into a foot muscle as in a previous trial — it appears safe and leads to production of the missing muscle protein dystrophin.

Big Horses, Big Time

"Pa, I’m gettin' darn tired of goin’ down there and comin’ back with second place."

Robert Powell, 14, ordinarily is a young man of very few words. But when the subject is horse-pulling contests, it garners the full attention of this lad from Pleasuresville, Ky.

New DMD Care Recommendations

A comprehensive set of clinical care recommendations for the diagnosis and management of Duchenne muscular dystrophy (DMD) is now available to medical professionals and families.

The complete recommendations,  in printable PDF format, can be read here:

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.

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.

LGMD Research: Modifier Gene

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.

Follistatin Genes Strengthen Muscles in Monkeys

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 diseases, as well as muscle damage due to other illnesses, injury or aging.

Pages