Quest Magazine

Possible New Therapy for OPMD?

Scientists in France and the Netherlands recently announced they've identified a promising new strategy that could potentially become a therapy for oculopharyngeal muscular dystrophy (OPMD), a form of MD that primarily weakens the eyelid and throat muscles and also can affect muscles in the limbs.

The strategy involves using an antibody (immune-system protein) derived from llamas. The antibody sticks to abnormally formed protein molecules in muscle cells and keeps them from forming large, damaging clumps.

MDA's Voice for Change

Palliative care for kids, transition services for young adults, and genetic discrimination are just some of the projects being worked on by MDA’s Advocacy program, under the energetic direction of MDA Vice President Annie Kennedy.

Following are some highlights of these projects.

Palliative care in pediatrics

DMD: Gene-Changing 'Cocktail'

Scientists at Children's National Medical Center in Washington, Carolinas Medical Center in Charlotte, N.C., and the National Center of Neurology and Psychiatry in Tokyo, have successfully treated dogs with a disease closely resembling Duchenne muscular dystrophy (DMD) , using a molecular treatment strategy called "exon skipping." The strategy is simultaneously under development in human patients.

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.

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