Inflammatory Myopathies

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 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.

Scientists at five U.S. institutions have successfully used gene therapy to improve muscle function in a single human subject with a hereditary form of inclusion-body myositis (IBM) caused by mutations of the GNE gene. Study results were published online March 30, in the Journal of Gene Medicine.

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.

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.

At least 15 Muscular Dystrophy Association research grantees, past and present, have won funding from the National Institutes of Health through the American Recovery and Revitalization Act (ARRA) to accelerate the search for treatments and cures for neuromuscular diseases.

Abnormal accumulation of a protein called "tau" has been considered by many to contribute to muscle degeneration in inclusion-body myositis (IBM). But recently, MDA grantee Steven Greenberg and colleagues at Brigham and Women's Hospital and Harvard Medical School in Boston have cast doubt on this purported disease mechanism and say it's too early to develop drugs for IBM based on it.

Abnormal accumulation of a protein called "tau" has been considered by many to contribute to muscle degeneration in inclusion-body myositis (IBM). But recently, MDA grantee Steven Greenberg and colleagues at Brigham and Women's Hospital and Harvard Medical School in Boston have cast doubt on this purported disease mechanism and say it's too early to develop drugs for IBM based on it.