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
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
Update (Feb. 2, 2015): Swiss pharmaceutical company Roche announced in a Jan. 16, 2015, press release, that it will acquire Trophos and continue development of olesoxime for SMA. "We will build on the work done by Trophos and the French Muscular Dystrophy Association to advance the development of olesoxime and to bring it to people who with live with this devastating condition as quickly as...
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
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
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
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
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
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...