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...
Researchers look at the 'window of opportunity' for effective treatment in spinal muscular atrophy and the therapeutic potential of celecoxib
Recent spinal muscular atrophy (SMA) research includes findings that shed additional light on the optimal "window of opportunity" for treatment and point toward a potential candidate for therapeutic development.
Isis Pharmaceuticals has launched a phase 2 trial to test its experimental antisense drug ISIS-SMNRx in infants with spinal muscular atrophy
Update (Nov. 4, 2013): All centers are now recruiting participants.
Riluzole and tirasemtiv — two drugs with connections to other neuromuscular disorders — are being tested as potential treatments for spinal muscular atrophy
In the search for therapies for spinal muscular atrophy (SMA), researchers are testing two drugs with connections to other neuromuscular disorders: riluzole, which is approved for use in amyotrophic lateral sclerosis (ALS); and tirasemtiv, which is already in testing for ALS and
Encouraging results were seen in an early-stage trial of ISIS-SMNRx, a drug designed to increase levels of a deficient protein in spinal muscular atrophy
An experimental drug designed to treat the underlying molecular defect in spinal muscular atrophy (SMA) has shown encouraging results in a phase 1 trial.
Gene therapy delivered to muscles reaches nerve cells; collaboration aims to advance gene therapy development program; a mouse model represents milder forms of the disease
Spinal muscular atrophy (SMA) is a disease in which nerve cells that control muscles (motor neurons) in the spinal cord die, causing progressive weakness in the voluntary muscles. Recent research news includes advances in delivery methods for gene therapy treatment of SMA, and creation of a new mouse model that could help scientists better understand and develop treatments for the disease....
Mice treated with the small-molecule compound improved muscle strength, increased motor neuron survival and boosted production of a motor-neuron support molecule called VEGF
Mice with a disorder mimicking human spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) that were treated with an experimental therapy called arimoclomol showed improved nerve-cell survival, increased body weight, and better muscle strength and function than mice that didn't receive the treatment.