Quest Magazine

Clinical Trial Info: Ceftriaxone in ALS

Researchers conducting a clinical trial of the antibiotic ceftriaxone in amyotrophic lateral sclerosis (ALS) currently are recruiting participants at 57 locations across the United States and Canada.

Becker MD Study Seeks Participants

A new study to determine the best "outcome measure" (measurable activity) with which to assess thigh-muscle (quadriceps) strength in men with Becker muscular dystrophy (BMD) is seeking participants.

The study, taking place at Nationwide Children's Hospital in Columbus, Ohio, is a necessary prelude to a planned trial of gene therapy involving injections of genes for the follistatin protein in people with BMD.

Experts Gather to Focus on Heart in DMD

Experts from around the world will gather Jan. 21-22, 2011, at an MDA-sponsored conference about the heart in Duchenne muscular dystrophy (DMD).

ALS Experts: Keep the Weight On

People with amyotrophic lateral sclerosis (ALS) should go ahead and eat foods high in fat and calories, but they also need to eat healthy foods as well, say experts on ALS nutrition who participated in an MDA-sponsored webinar on January 11, 2011.

MDA Awards $1.5 Million to Acceleron for DMD Drug Testing

MDA has begun funding tests of the experimental drug ACE-031 in children with Duchenne muscular dystrophy (DMD). The drug is being developed by Acceleron Pharma, a Cambridge, Mass., biotechnology company in collaboration with Shire, a global specialty biopharmaceutical company that focuses on developing, manufacturing and commercializing therapies for rare genetic diseases.

Nationwide Children's Podcast Explores Immunity in DMD

A January 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, probes a subject that's been on the minds of many researchers, doctors and families: autoimmunity (self-immunity) in Duchenne muscular dystrophy (DMD).

Gene Variant May Indicate Severity of DMD

A team of scientists working in the United States and Italy has uncovered a variant in the gene for a protein called osteopontin that appears to reliably indicate disease severity in most (but likely not all) cases of Duchenne muscular dystrophy (DMD).

The variant is apparently a genetic modifier of DMD, a disease in which the underlying cause is a mutation in the gene for the dystrophin protein and the resulting lack of dystrophin in the muscles.

‘CMD Standard of Care’ Guidelines Issued

Editor's note: This article was updated on Jan. 6, 2011, to include a direct, free link to the Journal of Child Neurology article about the CMD guidelines.

A panel of 82 international experts — including several MDA grantees and clinic directors — has produced the first-ever care guidelines for the congenital muscular dystrophies (CMD), a group of genetic neuromuscular disorders that have their onset at birth or in early infancy.

Ex-Con with SMA Now Mentors Children of Inmates

For eight long years, Terrence Stevens — who goes by the nickname "T-Wheels" — had the dubious distinction of being the only inmate in the New York state prison system with “muscular dystrophy.”

Stevens actually has spinal muscular atrophy (SMA) type 3, but he says no one in prison knew or cared what his true diagnosis was.

Conditions were horrible for inmates with disabilities, says Stevens, 43, who is nonambulatory and has only limited use of his hands.

Research Briefs: DMD, BMD, MMD, SMA

Editor's note 2/7/11: A link to the Tivorsan Pharmaceuticals website has been added.

Duchenne and Becker muscular dystrophies

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