Quest Magazine

Social Enterprise Business Sevenly Joins MDA in the Fight Against Muscle Disease

Weeklong Campaign to Sell Custom-Designed T-shirts
Inspired by the Strength of MDA Families and Supporters

CHICAGO, Nov. 24, 2014 — The Muscular Dystrophy Association  and, known for its seven-day, limited edition charity T-shirt campaigns, announced today they will join forces in the fight against muscle disease with the sale of five custom-designed T-shirts to benefit individuals and families served by MDA.

Phase 3 Trial of ISIS-SMNRx Now Open for Children with SMA

Update (Dec. 1, 2014): Isis has announced that its partner, Biogen Idec, plans to conduct a phase 2 trial of ISIS-SMNRx in up to 25 presymptomatic newborns genetically predisposed to develop SMA, as well as a phase 2 study of this drug in approximately 20 patients with infantile or childhood-onset SMA who do not meet the inclusion criteria for the current phase 3 studies. These two additional studies could begin in the first half of 2015.

PTC To Test RG7800 in SMA

PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, says it will soon open a trial to test the safety and tolerability of its investigational drug RG7800 in adults and children with spinal muscular atrophy (SMA). The announcement was made in a Nov. 19, 2014, press release.

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: