Motor Neuron

Research updates and clinical trials information

posted on October 30, 2014 - 9:01am
What About My Disease? Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.

A phase 1 gene transfer trial in nine babies with type 1 spinal muscular atrophy has opened at Nationwide Children's Hospital in Columbus, Ohio

posted on May 6, 2014 - 9:33am
A phase 1 trial to test the safety and efficacy of gene transfer therapy in infants with type 1 spinal muscular atrophy (SMA) who are 9 months old or younger has opened at Nationwide Children's Hospital in Columbus, Ohio, under the direction of neurologist Jerry Mendell.

Preliminary results for a phase 2 trial of the experimental compound olesoxime suggest it may preserve motor function in spinal muscular atrophy

posted on March 13, 2014 - 9:41am
Update (May 2, 2014): Trophos, developer of olesoxime to treat SMA, announced more detailed results from this trial at the spring 2014 annual meeting of the American Academy of Neurology and in an April 28, 2014, press release.

Preliminary results from four infants in a phase 2 trial of ISIS-SMNRx suggest the drug is well-tolerated and may prolong ventilator-free survival

posted on March 3, 2014 - 2:17pm
Interim results of a phase 2 clinical trial to test multiple doses of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA) suggest that the drug is well-tolerated and may prolong ventilator-free survival.

MDA-funded researchers say SMN, which is deficient in spinal muscular atrophy, is most needed during early life and in skeletal muscle and nerve tissues

posted on February 4, 2014 - 11:11am
Raising levels of the SMN protein, which is deficient in patients with the most common form of spinal muscular atrophy (SMA), has been the holy grail of therapy for this disease since the 1990s and is the goal of several experimental compounds now in development.

A spring 2014 course from Ohio State University will emphasize Duchenne muscular dystrophy mechanisms, research, and clinical and social aspects

posted on January 8, 2014 - 12:09pm
A course on Duchenne muscular dystrophy (DMD), geared toward graduate and upper-division undergraduate students and held at Ohio State University College of Medicine and Nationwide Children's Hospital in Columbus, is open to the public, without charge, via real-time streaming and podcasts.

A mouse model of adult-onset spinal muscular atrophy, developed with MDA support, suggests a treatment now in testing for early-onset disease may benefit adults

posted on September 17, 2013 - 2:25pm
Scientists supported in part by MDA have developed a new research mouse model with a disorder that mimics the adult-onset form of chromosome 5-related spinal muscular atrophy (SMA), a genetic disease of spinal nerve cells that control muscle activity (motor neurons).  

The quest to cure spinal muscular atrophy still has many years to go, but the signs of progress are unmistakable

posted on August 29, 2013 - 10:37am
A Closer Look: SMA Slideshow August is often referred to as Spinal Muscular Atrophy (SMA) Awareness Month, but for MDA, work on finding treatments for this degenerative disease — and providing assistance to individuals and families affected by it — is a year-round event. Today, research aimed at slowing, stopping or even preventing spinal muscular atrophy (SMA) is advancing with more speed...