Muscular Dystrophies

DMD/BMD: Global Trial Planned of Experimental Drug Ataluren

Trials of the experimental muscular dystrophy drug ataluren showed that the drug was generally well-tolerated and slowed the rate of decline in walking ability, compared to the placebo group.

After consulting with regulatory agencies in Europe and the United States, biopharmaceutical company PTC Therapeutics has decided to move ahead with a global, phase 3 confirmatory study of ataluren. Trial sites will be in the U.S. and at least 10 other countries.

DMD: Which Steroid Regimen Is Best?

Prednisone and other drugs in the corticosteroid family have become standard treatment for Duchenne muscular dystrophy (DMD) in much of the world for almost a decade. In this disorder, they slow the decline of muscle function and prolong walking ability. However, corticosteroids have significant side effects, such as weight gain, bone loss, cataracts and behavior changes.

Strong Community Support Essential to Passage of Critical Muscular Dystrophy Legislation

Note: The MD CARE Act is a critical part of the historic effort to find cures for muscular dystrophies. Watch for articles in the coming weeks that delve further into the remarkable progress attributable to this important legislation.

DMD: Vasodilator Drug Study Needs Four Boys

A 12-participant study of the acute effects of two vasodilating drugs on blood flow to exercising muscles needs four more boys with Duchenne muscular dystrophy (DMD) who meet study criteria and are able to travel to Los Angeles. Vasodilators increase the diameter of blood vessels.

$13.6 Million in New MDA Grants Promote Understanding, Treatment of Neuromuscular Diseases

The Muscular Dystrophy Association has awarded 44 new grants totaling $13.6 million to advance the understanding and treatment of neuromuscular diseases. The new grants, most of which took effect Feb. 1, encompass a range of diseases covered by MDA’s research program, and they support innovative approaches to basic research and new drug development.

In addition to addressing 16 specific neuromuscular diseases under MDA’s umbrella, the grants also fund research into muscular dystrophy in general, and research into muscle physiology related to neuromuscular disease.

LGMD2A Registry Seeks Participants

A recently opened global registry— a database of patient information — is seeking people with the type 2A form of limb-girdle muscular dystrophy (LGMD2A), a form of LGMD that results from a deficiency of the calpain 3 protein.

MDA Webinar Discusses Spinal Bracing and Surgery in Children

A one-hour, MDA-sponsored webinar features two physicians and the parent of a child with spinal muscular atrophy (SMA) who has undergone bracing and surgery for a spinal curvature, as well as questions and answers from listeners.

Muscle Physiology — Masahiro Iwamoto, Ph.D., D.D.S.

Masahiro Iwamoto, research scientist at the Children’s Hospital of Philadelphia and associate professor of pediatric orthopedics at the University of Pennsylvania School of Medicine, was awarded an MDA research grant totaling $405,000 over a period of three years to study new ways to reduce muscle degeneration.

MDA Establishes Myotonic Dystrophy Clinical Research Network

MDA has launched a new, five-center clinical research network focused on type 1 and type 2myotonic muscular dystrophy (MMD1 and MMD2, also known as DM1 and DM2), with the principal goal of preparing for testing of new MMD treatments as they become available.

DMD: Dystrophin-Deficient Dogs Benefit from Gene Therapy

For the first time, gene therapy using a highly miniaturized dystrophin gene resulted in significant improvement in muscle structure and function in dogs with a disorder mimicking human Duchenne muscular dystrophy.

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