Duchenne Muscular Dystrophy (DMD)

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.




MD Research: Muscle-Repair Booster

In experiments in mice, Michael Rudnicki, an MDA grantee at the Sprott Center for Stem Cell Research at Ottawa Hospital Research Institute (OHRI), and colleagues, found the WNT7a protein stimulates muscle repair by causing proliferation (an increase in number) of "satellite stem cells." They say the protein probably operates similarly in humans. The findings were published June 5, 2009, in the journal Cell Stem Cell.

Kids’ Respiratory Needs

The proceedings of a symposium titled "Pulmonary Management of Pediatric Patients with Neuromuscular Disorders" have been published as a supplement to the May 2009 issue of the journal Pediatrics.

The symposium was held Feb. 20, 2008, at Scottish Rite Hospital in Dallas, and was sponsored by MDA, as well as Respironics and Hill-Rom Services.

Topics, all of which pertain specifically to children with neuromuscular disorders, include

Utrophin Gets In

MDA grantee James Ervasti and colleagues at the University of Minnesota-Twin Cities in Minneapolis have found that a protein known as utrophin, injected into mice lacking the dystrophin protein and showing a disease resembling Duchenne muscular dystrophy (DMD), conferred significant benefits.

The experiments Ervasti and colleagues describe online May 26, 2009, in PLoS Medicine, are the first to show benefit from the direct injection into DMD mice of utrophin protein, rather than utrophin genes or gene modifiers.

Fighting the Fire

A protein called osteopontin has been implicated as a cause of some of the detrimental inflammation and scarring ("fibrosis") of muscle tissue that takes place in Duchenne muscular dystrophy (DMD). Eliminating osteopontin was beneficial to mice with a DMD-like disease, and the researchers concluded that reducing osteopontin should be investigated as a possible therapy for DMD.

DMD, MG Research: Spotlight on Prednisone

Some interesting findings about prednisone’s effect on behavior in DMD, and on drugs that may alter its usage in myasthenia gravis, were part of the 61st annual meeting of the American Academy of Neurology (AAN), held recently in Seattle.

DMD STUDY

Daily prednisone led to better behavior than weekly, high-dose prednisone

Biology Prize

On May 3, 2009, molecular biologist Louis Kunkel at Children's Hospital in Boston and Harvard University, and biophysicist Kevin Campbell at the University of Iowa, received the prestigious March of Dimes Prize in Developmental Biology. The prize includes a $250,000 cash award.

DMD: Restarting Muscle Development?

A protein called laminin 111 had a marked therapeutic effect in mice that lack the dystrophin protein and have a muscle disease resembling human Duchenne muscular dystrophy (DMD), say researchers at the University of Nevada School of Medicine.

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