Quest Magazine

FA Research: Idebenone Strikes Out Again

Idebenone, a substance believed to act as an antioxidant and to aid in energy production in cellular structures called mitochondria, has failed to benefit people with Friedreich's ataxia (FA) in a phase 3 trial conducted at several European centers. The drug is similar to coenzyme Q10, a naturally occurring molecule.

Pompe Disease: FDA Approves Lumizyme

The U.S. Food and Drug Administration (FDA) has approved U.S. sales of the enzyme-replacement drug Lumizyme, the first treatment in the U.S. specifically for late-onset Pompe disease.

Podcasts Focus on Latest Neuromuscular Research

Scientists at the Neuromuscular Disorders Program at Nationwide Children’s Hospital in Columbus, Ohio, are producing a series of podcasts examining current research in neuromuscular disease.

The series, “This Month in Muscular Dystrophy,” is an opportunity to hear authors of recent publications discuss how their work improves understanding of these diseases, and what it might mean for treatment.

Gene Therapy Success in IBM

Scientists at five U.S. institutions have successfully used gene therapy to improve muscle function in a single human subject with a hereditary form of inclusion-body myositis (IBM) caused by mutations of the GNE gene. Study results were published online March 30, in the Journal of Gene Medicine.

Scholarships for Caregivers

Four $2,500 scholarships — named in honor of a former MDA National Goodwill Ambassador — are being offered by the Rosalynn Carter Institute for Caregiving.  Deadline for applications is June 1.

The scholarships will provide financial assistance to family, professional, or paraprofessional caregivers of any age who are seeking training or education in specific skills, procedures and strategies that lead to more effective care and protect the health and well-being of the caregiver.

SOD1 Versus Other ALS: Apples and Oranges?

Scientists at Northwestern University Feinberg School of Medicine in Chicago have announced new findings showing some forms of familial and nonfamilial ("sporadic") amyotrophic lateral sclerosis (ALS) have commonalities at the molecular level — but that one form of familial ALS may be different.

DMD Research: New Funding for Imaging Studies

The use of magnetic resonance imaging (MRI) as an assessment tool in boys with Duchenne muscular dystrophy (DMD) is being studied by former MDA grantee Krista Vandenborne, through a $7.5 million grant from the National Institutes of Health (NIH).

The goal of the study is to assess whether MRI technology can be used as a precise, noninvasive measure of muscle tissue, gauging both disease progression and the effectiveness of therapies tested in children with DMD.

A Beach Park for EveryBODY

Designers of a new accessible beach park in Virginia Beach, Va., are very glad they asked Caroline Pennell, of Chesterfield, to look over their plans. The 14-year-old with limb girdle muscular dystrophy (LGMD) came up with the best idea of all. 

A Beach Park for EveryBODY

Designers of a new accessible beach park in Virginia Beach, Va., are very glad they asked Caroline Pennell, of Chesterfield, to look over their plans. The 14-year-old with limb girdle muscular dystrophy (LGMD) came up with the best idea of all. 

MTM: Unlocking Muscle Fibers to Myotubularin

MDA-supported researchers at 4s3 Bioscience, a biotechnology company in Medford, Mass., are using a new molecular strategy to transport a potentially therapeutic protein into muscles, as an experimental treatment for X-linked myotubular myopathy (MTM), a genetic muscle disease that's generally fatal in infancy.

The treatment will be tested in a mouse model of MTM. These mice do not make myotubularin and display signs and symptoms similar to those in humans with X-linked MTM.

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