Quest Magazine

ALS SOD1 Trial: A ‘Watershed Moment’

Isis Pharmaceuticals of Carlsbad, Calif., has begun a phase 1 clinical trial of its experimental compound ISIS-SOD1-Rx in people with familial (inherited) ALS caused by toxic SOD1 protein molecules.

Thinking of College? Think Scholarships

Application deadlines are fast approaching for two scholarships for students with disabilities, offered by two organizations run by people with forms of muscular dystrophy.

Both organizations – Incight and Deshae Lott Ministries -- require applicants to submit documentation of disability, confirmation of acceptance at a qualifying educational institution, and letters of recommendation. More details are available on their Web sites.

Incight
(971) 244-0305
www.incight.org

DMD/BMD Research: Ataluren Results Disappointing

The biopharmaceutical firm PTC Therapeutics announced March 3 that ataluren, its experimental drug for certain forms of Duchenne (DMD) and Becker (BMD) muscular dystrophy, although safe and well tolerated, failed to meet its primary end point within the 48-week duration of the phase 2b trial. That end point was an improvement in how far boys with DMD or BMD could walk in six minutes.

Gene Therapy Rescues Mice with SMA

Scientists at four U.S. institutions have successfully used gene therapy to treat very young mice with a disease resembling severe spinal muscular atrophy (SMA).  Study results were published online Feb. 28, 2010, in the journal Nature Biotechnology.

A Quicker Trip from ‘Microscope to Marketplace’?

The National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA) have announced plans to establish a Joint NIH-FDA Leadership Council, with the goal of shortening the time it takes to move promising therapies through the regulatory process and into the hands of waiting patients.

SMA Therapeutics: Changing the Code

 A process called trans-splicing has been shown to increase levels of a needed protein in mice with a disease resembling severe human spinal muscular atrophy (SMA), says a research team at the University of Missouri-Columbia, whose findings were published Jan. 6, 2010, in the Journal of Neuroscience.

The Play’s the Thing

Surrounded by loaves of bread, 12-year-old Andrew Longwell looks like a real baker. The chef’s hat helps, too.

The sixth-grader from Rochester, N.Y., completes the picture with his voice. He sings a charmingly youthful rendition of “Who Will Buy?” one of the songs in the musical “Oliver!” He and other students are performing the show at the Greece Athena Middle School Theatre in Rochester for two weeks starting Feb. 26.

Wasn’t gonna do it

Building New Muscle Requires Controlled Damage

Remodeling a building usually requires some degree of dismantling before new construction can begin. That principle, it now seems, also may apply to the remodeling of the body's cells. 

Scientists at Ottawa Hospital Research Institute and the University of Ottawa have found recently that it applies to muscle cells, which can only develop from a stemlike state into mature muscle fibers after a certain amount of their DNA has been disassembled and then rebuilt. 

About the new findings 

SMA Research: Strengthening the Junctions

MDA-supported researchers at the University of Ottawa and Ottawa Hospital Research Institute (OHRI) have identified a biological pathway that may prove useful in developing treatments for spinal muscular atrophy (SMA)

Causative Gene Mutations ID'd for Two Muscle Diseases

An MDA-supported, multinational team of researchers from Canada and Europe has identified specific mutations in the anoctamin 5 (ANO5) gene on chromosome 11 that can cause type 2L limb-girdle muscular dystrophy (LGMD2L) and type 3 Miyoshi myopathy.

Pages