Duchenne Muscular Dystrophy (DMD)

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.

DMD Research: Diagnostic Delays Common

A study that analyzed medical records from four U.S. states has found that the average time between symptom onset and diagnosis of Duchenne muscular dystrophy (DMD) is 2.5 years, an interval that hasn't changed in two decades.

This delay in identification postpones treatment that can slow the progression of the disease and results in lost opportunities for genetic counseling of parents.

MD Research: 3-Protein Repair Cluster

Scientists in the United States and Japan have identified a three-protein cluster that reseals damaged muscle-fiber membranes. The findings, published June 5, 2009, in the Journal of Biological Chemistry, could have implications for development of treatments for muscular dystrophies.

Three-Protein Repair Cluster Identified

Scientists in the United States and Japan have identified a three-protein cluster that reseals damaged muscle-fiber membranes. The findings, published June 5, 2009, in the Journal of Biological Chemistry, could have implications for development of treatments for muscular dystrophies.




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