Quest Magazine

ALS Fatigue Fighter

Study results reported in the March 2009 issue of Muscle & Nerve indicate that modafinil (marketed under the brand name Provigil) "may be a promising intervention for fatigue in ALS (amyotrophic lateral sclerosis) patients." Fatigue and daytime sleepiness often accompany this disease.

Hiroshi Mitsumoto, director of the Eleanor and Lou Gehrig MDA/ALS Research Center at Columbia University Medical Center in New York, where the study was based, received MDA support to conduct it.

Building a Better Gene

Displacement of a protein called neuronal nitric oxide synthase (nNOS) from the membrane that surrounds each skeletal muscle fiber appears to be a much more important contributor to exercise intolerance and even cardiac degeneration in some forms of muscular dystrophy than previously recognized.

DMD Research: Exon Skipping

On Jan. 21, AVI BioPharma of Portland, Ore., announced its experimental compound AVI4658 for the treatment of Duchenne muscular dystrophy (DMD) yielded promising results in a phase 1 clinical trial in the United Kingdom.

Experts Discuss ALS

Nearly 600 conferees gathered in Las Vegas Jan. 25-28 for the 2009 MDA National Clinic Directors Conference, where a number of experts presented a broad range of topics covering neuromuscular disease.

One particular area of focus was ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease). Highlights included discussions of cognitive difficulties, immunization strategies in certain of the familial forms of the disease, and stem cells as possible therapy.

Experts Discuss ALS

Nearly 600 conferees gathered in Las Vegas Jan. 25-28 for the 2009 MDA National Clinic Directors Conference, where a number of experts presented a broad range of topics covering neuromuscular disease.

One particular area of focus was ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease). Highlights included discussions of cognitive difficulties, immunization strategies in certain of the familial forms of the disease, and stem cells as possible therapy.

Abnormal behavior in ALS may deserve closer look

DMD Research: Exon Skipping Goes Systemic

AVI BioPharma of Portland, Ore., has started the systemic (through the blood) delivery phase of its clinical trial of AVI4658 in Duchenne muscular dystrophy (DMD). The trial is being conducted in the United Kingdom.

Mutations in FUS Gene are a Cause of Familial ALS

Two independent research teams, one based in the United States and Canada and the other in the United Kingdom and Australia, have identified mutations in a gene called FUS on chromosome 16 as a cause of familial amyotrophic lateral sclerosis (ALS).

Both groups announced their findings in the Feb. 27, 2009, issue of the journal Science.

Stem Cell Research: Major Step

The U.S. Food and Drug Administration (FDA) has said yes to a small safety trial of nervous-system stem cells in people with recently sustained injuries to the middle (thoracic) part of the spinal cord, the biopharmaceutical company Geron Corporation of Menlo Park, Calif., announced Jan. 23, 2009.

Dogs with ALS

Researchers at several institutions in the United States and Sweden have found that a mutation in the gene for superoxide dismutase 1 (SOD1), known to cause ALS (amyotrophic lateral sclerosis) in 1 percent to 3 percent of human cases, also can cause an ALS-like disease in dogs.

These dogs are the first spontaneously occurring animal model of ALS discovered, the researchers say in their paper, published online Feb. 2 in Proceedings of the National Academy of Sciences.

Stem Cell Research: Major Step

The U.S. Food and Drug Administration (FDA) has said yes to a small safety trial of nervous-system stem cells in people with recently sustained injuries to the middle (thoracic) part of the spinal cord, the biopharmaceutical company Geron Corporation of Menlo Park, Calif., announced Jan. 23, 2009.

Pages