Quest Magazine

New Phase 2b Trial of Tirasemtiv (CK-2017357) in ALS

Update (Oct. 29, 2012): This article has been updated to reflect the fact that the trial is now open to recruitment.

MDA Muscle Symposium: Newborn Screening in DMD

Update (Feb. 8, 2013): Longtime MDA grantee Jerry Mendell, who directs the Center for Gene therapy at Nationwide Children's Hospital in Columbus, Ohio, published Report of MDA Muscle Disease Symposium on Newborn Screening for Duchenne Muscular Dystrophy in Muscle & Nerve, Feb. 8, 2013.

NINDS Launches Clinical Trial Network, SMA Biomarkers Study

Update (March 28, 2013): This story was updated with a link to the ClinicalTrials.gov site for this study.

DMD Briefs: Stopping Inflammation on Earth and in Space

Inflammation— the immune system's first line of defense in tissue that's been damaged by injury or infection — is a good example of a process that's a good thing up to a point and under certain circumstances and a bad thing in excess or under the wrong circumstances.

IGF1 Shows Benefit in SBMA Mice

Mice with a disease resembling spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) that were treated with a compound based on insulin-like growth factor 1 (IGF1) had better motor function, slower weight loss, healthier muscles and longer survival time than mice that received an inactive substance, an MDA-supported research team has reported.

BMD Conference Videos Cover Health Care, Research and Daily Living

Videos of the entire MDA-sponsored Becker Muscular Dystrophy Conference, held in Chicago on Aug. 11, 2012, are now archived on the Conference video page.

ALS Risk Elevated in Some Retired NFL Players

Update (Nov. 6, 2012):This story was updated to reflect the availability of an abstract of the scientific paper (online and print) on which the story is based, as well as information about an upcoming webinar on this subject.

ALS and SMA: Less EPHA4 Means More Motor Neurons Survive

Decreased activity of the EPHA4 receptor, which is encoded by the EPHA4 gene, increases life span in people with amyotrophic lateral sclerosis (ALS), a team of scientists has reported.

The receptor, which was found to modify disease in zebrafish, mice, rats and humans, works together with a gene recently associated with ALS — profilin 1 (PFN1).  

Sarepta Issues ‘Open Letter’ Explaining Development of Eteplirsen for DMD

In July 2012, Sarepta Therapeutics announced encouraging interim results for its phase 2b trial of eteplirsen, an experimental exon-skipping drug in development to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene.

Chemical 'Signature' May Be Used to Track ALS

A "pro-inflammatory" chemical signature displayed by monocytes (a type of white blood cell) appears to signal the presence of amyotrophic lateral sclerosis (ALS) even before symptoms begin, a team of scientists has reported. If verified, the blood biomarker may make it possible for physicians to monitor disease progression using a simple blood test.

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