Quest Magazine

ALS Registry Touts Revamped Website, New Surveys

In hopes of collecting as much information as possible, the National ALS Registry, which opened in October 2010, already is expanding its reach.

Registry officials have made the enrollment process easier and are adding surveys about potential risk factors to the registry’s website.

Research Briefs: FA, MG, MM, MMD1, gene therapy

Edison drugs target FA, mitochondrial diseases

Second Phase 2 Trial of CK-2017357 Under Way

San Francisco biotechnology company Cytokinetics announced June 21, 2011, that it has opened enrollment for a new clinical trial of CK-2017357 at three different dosage levels in people with ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

WWE and MDA to Recognize Youth Volunteers

Do you know an exceptional kid between the age of 6 and 17 who has volunteered for MDA or supported someone with a neuromuscular disease? Here’s your chance to nominate him or her for some well-deserved recognition.

WWE (World Wrestling Entertainment) and MDA are looking for two Community Champions this summer: young people who have demonstrated “exceptional commitment” to supporting MDA or who have made a “significant contribution” to an individual with muscle disease within the last two years.

ALS Research Briefs: Ataxin 2 Gene

In a 2010 study, scientists working in the United States and Germany found that small polyglutamine ("polyQ")expansions of DNA in the ataxin 2gene significantly increase the risk for developing ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

Expansions of DNA coding for glutamine in the ataxin 2 gene lead to production of expanded ataxin 2 proteins, containing extra strings of glutamine amino acid molecules.

Stem Cell Model Creates 'ALS in a Dish'

A human cellular model of ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease) has been created using cells taken from people with a subtype of the disease called ALS8.

This so-called “disease in a dish” research model already is enabling researchers to learn more about the role of a protein called VAPB in the ALS disease process.

Researchers Studying DMD-Affected Families

Belen Pappa, a graduate student in genetic counseling, is seeking participants for a survey-based study about psychosocial functioning in families in which a child has Duchenne muscular dystrophy (DMD).

Safety Stem Cell Trial in ALS Set to Advance

Maryland biotherapeutics company Neuralstem has announced favorable results from the first part of a phase 1 trial to test the safety of its experimental spinal cord stem cell treatment in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

Research Briefs: Stem Cells

Stem cells are a hot topic these days in medicine, science and law, although the term has multiple meanings and it's easy to get confused.

In short, stem cells are cells at an early stage of development from which specialized cells, such as muscle or nerve cells, can develop (in other words, from which these specialized cells "stem").

Different kinds of stem cells are referred to as:

Research Briefs: The DMD/BMD-Affected Heart

In both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), deterioration of the heart muscle, a condition known as cardiomyopathy, is a major cause of disability and death.

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