Quest Magazine

PTC Begins Submission of New Drug Application for DMD Stop Codon Read-Through Drug

New Jersey-based PTC Therapeutics has begun submitting a rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of its experimental drug for the treatment of Duchenne muscular dystrophy (DMD) caused by specific flaws (mutations) in the dystrophin gene.

Myostatin Blocker To Be Tested in DMD

Multinational pharmaceutical comany Pfizer recently opened a phase 2 study of an experimental compound that may be beneficial in Duchenne muscular dystrophy (DMD) through its ability to block myostatin, a naturally occurring protein that is known to limit muscle growth.

DMD: Utrophin Modulator SMT C1100 To Undergo Further Testing

SMT C1100, an experimental compound designed to boost levels of the potentially therapeutic protein utrophin in patients with Duchenne muscular dystrophy (DMD), will undergo further testing in boys with this disorder along with dietary specifications that may improve its absorption by the body, its developer has announced.

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

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