Quest Magazine

BMD: Follistatin Gene Transfer Results Encouraging

Results from a trial involving injection of genes for the follistatin protein into the thigh muscles of both legs in six men with Becker muscular dystrophy (BMD) show the experimental treatment appears safe and and was associated with improvement in walking ability (distance walked in six minutes) in four of the six trial participants.

BioBlast Is Developing an OPMD Drug

Israeli biotechnology company BioBlast Pharma is developing an experimental drug designed to treat oculopharyngeal muscular dystrophy (OPMD) and is testing it in a phase 2-3 clinical trial at sites in Jerusalem and Montreal. If the U.S. Food and Drug Administration (FDA) approves the proposal, the company plans to open a trial site in the Los Angeles area of the U.S.

AveXis SMA Gene Therapy Trial Continues Recruiting

Dallas-based biotechnology company AveXis, which is developing gene therapy for spinal muscular atrophy (SMA), announced in October that administration of its experimental gene transfer compound to the first three patients in a

Drug Development for DMD: Fall 2014 Update

Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014.

PTC is moving forward with ataluren

AAN Releases Guideline for LGMD Diagnosis and Care

Recommendations for diagnostic procedures or care were rated by the AAN panel according to the strength of the recommendation. The AAN uses the following rating scale:

Sarepta Announces Revised Timeline for Eteplirsen FDA Application

Boys with Duchenne muscular dystrophy between the ages of seven and 16 are now being recruited to participate in a phase 3 clinical trial to evaluate the effectiveness and safety of eteplirsen in treating DMD.

SMA: Further Data on ISIS-SMNRx Shows Promising Results

Isis Pharmaceuticals, developer of the experimental spinal muscular atrophy (SMA) drug ISIS-SMNRx, revealed positive results from phase 2 clinical studies of the drug focusing on infants and children with SMA. The new data, which was shared on October 10 by ISIS representatives attending the 19th International World Muscle Society Congress in Berlin, included the following highlights:

LEMS: Firdapse Study Shows Encouraging Results

Catalyst Pharmaceuticals, a biopharmaceutical company focused on rare debilitating diseases, announced encouraging results on Sept. 29, 2014, from its phase 3 clinical trial of Firdapse (amifampridine phosphate tablets equivalent to 10mg amifampridine) for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS).

MDA Team Momentum Fights Back Against Muscle Disease at The 2014 Bank of...


CHICAGO, Oct. 2, 2014 — The Muscular Dystrophy Association's new nationwide endurance program MDA Team Momentum will take huge strides in the fight for muscle health at the 2014 Bank of America Chicago Marathon  on Sunday, Oct. 12  to help support MDA's mission to save and improve lives of people fighting muscle disease.

MDA Names Dr. Grace Pavlath to Head Scientific Program as Research Department Intensifies Focus on Ushering Treatments Through Drug Pipeline