Quest Magazine

Drug Development for DMD: February 2015 Update

Development of treatments for Duchenne muscular dystrophy (DMD) continues to advance. Many of the new investigational drugs are potentially applicable to all DMD patients, while a few target those with specific mutations in the dystrophin gene.

Fighting fibrosis

Dear ALS Community

Sailormen Inc. Kicks Off Annual "Appetite for a Cure" Campaign to Help MDA Fuel the Fight for Muscle Health

Genervon's 'Master Regulator' Drug Encourages ALS Community

Biopharmaceutical company Genervon has announced what it considers encouraging  results from a "compassionate use" trial of its experimental drug GM604 in a single patient with advanced  amyotrophic lateral sclerosis (ALS).

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.