Duchenne Muscular Dystrophy (DMD)

First Human Exon Skipping Trial in US Planned for 2010

DMD Research: 'Remarkable' Results

Mice with a severe disease closely resembling human Duchenne muscular dystrophy (DMD) have responded extremely well to a new "exon skipping" compound that targets the specific dystrophin gene error these mice have.

Researchers noted "a remarkable prevention of the dystrophic pathology and improvement of the muscle function" in these severely affected mice, which lack both the dystrophin and utrophin muscle proteins. (Mice in previous exon skipping trials lacked only dystrophin, the protein missing in DMD.)

A Promising New Development for Treatment of Duchenne Muscular Dystrophy

A technique called exon skipping shows great potential to increase muscle strength and prolong life in people with a severe form of Duchenne muscular dystrophy (DMD). According to a study that included MDA-supported Stephen Wilton at the University of Western Australia, exon skipping improves production of a crucial muscle protein that’s missing in people with DMD. For the first time, these results were observed in mice with an especially severe form of muscular dystrophy.

Exon Skipping Proves Effective in Mice with a Severe DMD-Like Disease




Stephen Wilton

DMD Research: Drug Company On Board

On Oct. 13, 2009, the multinational pharmaceutical company GlaxoSmithKline announced it will develop and commercialize the experimental “exon skipping” compound PRO051 for Duchenne muscular dystrophy (DMD).

The commitment of a major pharmaceutical company to development of a drug for DMD is very good news for families with this disease.

Major Pharmaceutical Company Commits to Develop Exon Skipping Drug

On Oct. 13, 2009, the multinational pharmaceutical company GlaxoSmithKline announced it will develop and commercialize the experimental “exon skipping” compound PRO051 for Duchenne muscular dystrophy (DMD).

The commitment of a major pharmaceutical company to development of a drug for DMD is very good news for families with this disease.

Major Pharmaceutical Company Commits to Develop Exon-Skipping Drug

On Oct. 13, 2009, the multinational pharmaceutical company GlaxoSmithKline announced it will develop and commercialize the experimental “exon-skipping” compound PRO051 for Duchenne muscular dystrophy (DMD).

The commitment of a major pharmaceutical company to development of a drug for DMD is very good news for families with this disease.

DMD Idebenone Trial

This story was updated 11/9/10 with contact information for this trial and information about clinical trials in general.

Pediatric Neurologist Richard Finkel, co-director of the MDA neuromuscular disease clinic at Children's Hospital of Philadelphia, will be the lead investigator for the North American arm of a large international trial with idebenone in Duchenne muscular dystrophy (DMD).

DMD, BMD Research: Utrophin from Obesity Drug?

An experimental drug being developed to treat obesity and high blood lipid (fat) levels also may have promise for the treatment of Duchenne (DMD) and Becker (BMD) muscular dystrophies, according to new, MDA-supported research from the University of Ottawa.

MDA grantee Bernard Jasmin and graduate student Pedro Miura coordinated and led the study team, which published results online Sept. 10, 2009, in Human Molecular Genetics.

Obesity Drug Increases Utrophin

An experimental drug being developed to treat obesity and high blood lipid (fat) levels also may have promise for the treatment of Duchenne (DMD) and Becker (BMD) muscular dystrophies, according to new, MDA-supported research from the University of Ottawa.

MDA grantee Bernard Jasmin and graduate student Pedro Miura coordinated and led the study team, which published results online Sept. 10, 2009, in Human Molecular Genetics.

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