Quest Magazine

First US Exon-Skipping Trial Opens

Update (Nov. 5, 2012): Results announced in November 2012 showed the drug, now called drisapersen, reached blood levels approximately proportional to the injected dose at two of the three dosage levels tested and was not associated with serious adverse events. See Drisapersen Appears Safe in Non-Walking Boys With DMD.

It's Myasthenia Gravis Awareness Month

In recognition of June being National Myasthenia Gravis Awareness Month, Quest News Online is examining the disease from several perspectives. This article offers an overview of signs, symptoms, diagnosis and treatments for myasthenia gravis (MG), as well as some drugs for people with MG to avoid.

Augie's New Book Extols Giving

When MDA ALS Division Co-Chair Augie Nieto was a young businessman just starting out, he had an idea that laid the groundwork for his later, multimillion-dollar success in the fitness industry. Instead of selling his unwanted exercise bikes at a loss, he sent 50 of them as gifts to health club owners across the country. Soon, orders started pouring in.

The Flip of a Switch

Neuraltus Pharmaceuticals of Palo Alto, Calif., is developing a small molecule whose target is regulation of immune system cells believed to contribute to neuroinflammation and disease progression in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

About ALS mechanisms  

The Rap on MD: Teen with CMD releases album

Eighteen-year-old Austin Puckett — Puck — is an up-and-coming hip-hop (rap) artist from Waynesville, Ohio, who uses his experiences with congenital muscular dystrophy as inspiration for his musical career.

Puckett has released two albums, “Million Dollar Dreams” and “Still Wishing,” on which he both wrote and performed the lyrics. ReFraze Studios in Dayton handled recording of both. The albums convey upbeat messages about the challenges in Puckett’s life — with none of the violence or vulgarities often associated with rap.

ALS Ceftriaxone Trial Still Open

A 600-person trial of intravenous ceftriaxone, an antibiotic in the cephalosporin family that's approved to treat certain types of infections, is still looking for participants at 53 sites in the United States and Canada.

About the trial

Laboratory studies have suggested that ceftriaxone protects motor neurons (the cells that die in ALS) from injury.

To be included in the study, participants must:

OPMD: Cystamine Strengthens Muscles in Mice

Scientists in the United Kingdom have found that mice carrying a genetic mutation that causes oculpharyngeal muscular dystrophy (OPMD) in humans and showing a disease resembling human OPMD benefited from treatment with a chemical called cystamine, provided in their drinking water.

About the new findings

David Rubinsztein and colleagues at the University of Cambridge announced their findings June 2, 2010, in Science Translational Medicine.

DMD Trial: AVI4658 Increased Dystrophin Production

The biopharmaceutical company AVI BioPharma has announced additional encouraging results from its clinical trial of AVI4658, an experimental treatment for Duchenne muscular dystrophy (DMD).

The new results show that, at higher doses, AVI4658 can result in substantial production of the needed dystrophin protein in muscle fibers.

The company has not yet released results of any tests of muscle function in the 19 children in this trial.

ALS TDI: Changes in the Works

In its quarterly Research Update Webcast on May 18, 2010, the ALS Therapy Development Institute (ALS TDI) informed viewers of a "paradigm shift" in TDI research. The changes include the addition of a new mouse model to the Institute's preclinical drug development program and an increased emphasis on the importance of biological indicators called "biomarkers."

 The webinar also addressed the current state of drug development in ALS, and the ways in which ALS TDI hopes to hasten the process.

Comedian with EDMD Takes Sacramento by Storm

 What started out as an item on O'Connell's bucket list is now an almost nightly occurance: performing comedy on stage.

“So this woman comes up and tries to give me a dollar. I wanted to say, ‘Hey! Just because I’m in a wheelchair doesn’t mean I’m homeless!’”

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