Quest Magazine

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.

Tom Mumper: Wood Turner with CMT

Tom Mumper
Tom Mumper, 80, revels in shaping wood into “functional art

Twenty years and 4,600 artfully crafted pieces later, Tom Mumper has no plans to slow down with his unique woodturning avocation.

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.

CMT Research Network

In January, MDA began funding development of the North American CMT Network to provide an infrastructure for clinical research in Charcot-Marie-Tooth disease (CMT) and aid researchers in locating potential participants for clinical studies.

An early goal of the network is to establish scoring systems for functional evaluations in children with CMT.

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