Quest Magazine

Trial of Antisense Drug for Type 1 Myotonic Dystrophy Opens

A phase 1-2 trial of ISIS-DMPKRx, an experimental compound for the treatment of type 1 myotonic muscular dystrophy, is now underway in the U.S. in adults with this disorder who are 20 to 55 years old and meet other study criteria.

MDA-Endorsed ABLE Act Passes

UPDATE: PRESIDENT SIGNS ACHIEVING A BETTER LIFE EXPERIENCE (ABLE) ACT INTO LAW, DECEMBER, 19, 2014

Bill Secures Tax-Deferred Savings Accounts For Those With Disabilities

MDA Community Helps Pass Newborn Screening Saves Lives Reauthorization Act

UPDATE: PRESIDENT SIGNS NEWBORN SCREENING SAVES LIVES REAUTHORIZATION ACT INTO LAW, DECEMBER, 19, 2014

MDA says bill could lead to earlier diagnoses, better treatment outcomes for people fighting certain muscle diseases

Drug Development for DMD: Fall 2014 Update-Part 2

Several experimental drugs are i development to treat Duchenne muscular dystrophy (DMD), a genetic disorder that results in a lack of the dystrophin protein in cardiac and skeletal muscle cells.

The ALS Association and MDA Team Up to Advance ALS Therapy Concept

Major League Baseball’s Commitment to Fighting ALS and

The ALS Ice Bucket Challenge Spurs Funding

Sarepta Reaffirms Commitment to Eteplirsen, Other DMD Drugs

Cambridge, Mass.-based Sarepta Therapeutics, developer of eteplirsen and other "exon-skipping" drugs designed  to treat Duchenne muscular dystrophy (DMD), provided overall reassurance about eteplirsen's development and some details of future plans to the DMD community in a Dec.

ALS: Mexiletine Helped Muscle Cramps But Did Not Show Other Benefits

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 Sevenly.org, 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.

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