Quest Magazine

Global CMD1C/LGMD2I Registry Opens

A new registry has been launched for people with conditions caused by mutations in the gene for fukutin-related protein (FKRP). This includes people with the type 2I form of limb-girdle muscular dystrophy (LGMD) and the type 1C form of congenital muscular dystrophy (CMD) and, in rare instances, the congenital muscular dystrophies muscle-eye-brain disease and Walker-Warburg syndrome.

Families and Experts Gather at 2011 BMD Conference

Community and empowerment were the themes of the third annual Becker Muscular Dystrophy (BMD) conference held at Cedars-Sinai Medical Center in Los Angeles on August 13, 2011.

Some 150 in-person attendees, and at least that many online attenders, engaged physicians, researchers, physical therapists and other BMD experts on a variety of topics, including the latest advances in research, medical management, physical therapy and clinical trials.

DMD: Phase 2 Exon-Skipping Trial Launched

Updated: View MDA's video about the launch of the eteplirsen trial on YouTube.

'Milk-Making' Molecule Lengthens Life Span in SMA Mice

The administration of a protein called prolactin has been shown to slow weight loss, improve motor function and increase life span by approximately 70 percent in mice with a disease resembling a severe form of spinal muscular atrophy (SMA).

Prolactin is a hormone whose primary function is to promote milk production in women who are breastfeeding.

SMA: Wider ‘Window of Opportunity’ for Treatment?

The "window of opportunity" for treating infants at risk of developing spinal muscular atrophy (SMA) may not be as narrow as some experts have feared, new experiments in mice suggest.

'Inhibitor' Molecule Helps ALS Mice be Stronger, Live Longer

An experimental compound called trichostatin A (TSA) has been shown to improve nerve-cell survival, increase strength and muscle weight, and result in longer life span in mice with a disease resembling ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

Eteplirsen (AVI-4658) Boosts Dystrophin Production in DMD

Complete and extremely encouraging findings from a phase 1b-2 trial of eteplirsen (AVI-4658), an exon skipping drug in development to treat a portion of the Duchenne muscular dystrophy (DMD) population, show the compound is safe and well-tolerated, and that it can significantly increase production of the needed dystrophin protein in recipients without eliciting an unwanted immune response.

BrainStorm Hopes to Bring ALS Stem Cell Trial to US

Biotechnology company BrainStorm Cell Therapeutics has announced plans to collaborate with two American institutions to test its experimental stem cell technology in people with amyotrophic lateral sclerosis (ALS) in the United States. The institutions are Massachusetts General Hospital in Boston and the University of Massachusetts Medical School in Worcester, Mass.

DMD: Daily, Weekly Prednisone Treatment Results About the Same

A one-year, MDA-supported study comparing a weekend-only prednisone treatment schedule with a daily prednisone schedule in boys with Duchenne muscular dystrophy (DMD) has found that the two treatment regimens provide about the same benefits and have approximately the same side-effect profile.

ALS Registry Touts Revamped Website, New Surveys

In hopes of collecting as much information as possible, the National ALS Registry, which opened in October 2010, already is expanding its reach.

Registry officials have made the enrollment process easier and are adding surveys about potential risk factors to the registry’s website.

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