Quest Magazine

DMD: Prosensa, GSK Expand Exon-Skipping Program

Update (Feb. 1, 2013): Dutch biopharmaceutical company Prosensa announced Jan. 29 that it has received orphan drug status in the United States and the European Union for compounds in development for the treatment of Duchenne muscular dystrophy.

ALS: SOD1 Protein Can Cause Trouble Even When It's 'Normal'

It's been known since the early 1990s that mutations in the superoxide dismutase-1 (SOD1) gene can result in the production of any number of varieties of improperly folded (misfolded) SOD1 protein, and that these misfolded proteins can cause familial amyotrophic lateral sclerosis (ALS).

SMA: Participants Sought for Study About Recreational Activities

Investigators at Northwestern University in Chicago are seeking people with spinal muscular atrophy (SMA), ages 2 to 21, to participate in a study about preferences and involvement in recreational and leisure activities.  

'Guided Missile' Strategy for MG Shows Promise in Mice

Researchers funded in part by MDA say a gene-based therapy designed to treat myasthenia gravis (MG) has shown promise in mice with an MG-like disease.

The research team was led by Dan Drachman, a longtime MDA research grantee at Johns Hopkins University in Baltimore, who also co-directs the MDA neuromuscular disease clinic at that institution. Drachman, a professor of neurology, has a special interest in MG, which is an autoimmune neuromuscular disease.

ALS Researcher Elected to Institute of Medicine

Don Cleveland, a longtime MDA research grantee studying amyotrophic lateral sclerosis (ALS), has been elected to the Institute of Medicine (IOM). Election to the IOM is one of the highest honors in the fields of health and medicine, and recognizes individuals who have demonstrated outstanding professional achievement and commitment to science.

Anti-Cancer Drugs May Help Build Muscle

Experimental anti-cancer drugs that block IAP (inhibitor of apoptosis) genes and kill cancer cells have unexpectedly been found to induce the growth and repair of muscle tissue, say scientists supported in part by MDA.

Ventricular Assist Device Implanted in Man with Duchenne MD

A left ventricular assist device (LVAD), which helps the heart pump blood throughout the body, was implanted in the chest of a young man with Duchenne muscular dystrophy (DMD) at Cincinnati Children's Hospital Medical Center in September. The patient is 29-year-old Jason Williams, of Peebles, Ohio.

Nationwide Children’s Podcast Explores the Congenital Muscular Dystrophies

Editor's note (Oct. 19, 2012): This story was updated to reflect the fact that Kevin Flanigan co-directs the MDA Clinic at Nationwide Children's Hospital in Columbus, Ohio.

Interim Results Suggest Chaperone Drug Enhances Enzyme Treatment in Pompe Disease

Biopharmaceutical company Amicus Therapeutics presented updated and encouraging results for its experimental Pompe disease (acid maltase deficiency) compound AT2220 this week at the 17th International Congress of the World Muscle Society in Perth, Australia.

Neural Stem Cell Trial in ALS ‘Very Successful’ So Far

Results from a completed phase 1 trial of neural stem cells in people with amyotrophic lateral sclerosis (ALS) show that the stem cells and the surgical method used to transplant them were safe and well-tolerated. In addition, the experimental therapy appears to have "interrupted the progression of the disease" in some trial participants.

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