Quest Magazine

MDA Names Kristin Stephenson to VP of Policy and Advocacy to Build on Recent Landmark Victories

DMD: Eteplirsen Data Still Encouraging After Three Years

Boys with Duchenne muscular dystrophy (DMD) caused by specific genetic mutations who were treated with the experimental drug eteplirsen for 168 weeks (3.2 years) continued to show an advantage in the distance they were able to walk compared to those treated with the drug for only 144 weeks (2.8 years), although all boys had declined in walking distance since the start of this phase 2b trial of eteplirsen.

ALS Stem Cell Trials Move Forward

Stem cells -- cells at an early stage of development that can mature and fulfill specific roles depending on their environment and signals they receive – have intrigued scientists as a possible treatment for amyotrophic lateral sclerosis (ALS) for several years. Now, two biotechnology companies – BrainStorm Cell Therapeutics and Neuralstem – are reporting encouraging results from ALS clinical trials.

Wisconsin Girl Returns as MDA National Goodwill Ambassador in 2015

Stanford Biobank Seeks Tissue Samples

Ever wondered how someone with a neuromuscular disorder in his or her family might contribute to research efforts in this field?

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

Pages