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.
An informational video describes an ongoing National Institutes of Health study of natural history and biomarkers in spinal muscular atrophy
Update (March 28, 2013): This story was updated with a link to the ClinicalTrials.gov site for this study.
Researchers conducting a study of natural history and biomarkers in infants with type 1 spinal muscular atrophy (SMA) currently are recruiting participants at 15 locations across the United States.
In the past, children with rapidly progressive neuromuscular scoliosis often underwent growth-stopping spinal fusion. Today, there are more options.
Payton Mueller's parents knew something was amiss by the time he was 9 months old.
Repligen’s spinal muscular atrophy program, including development of the experimental drug RG3039, now has the support of a major pharmaceutical company
The biotechnology firm Repligen today announced it has entered into an agreement with the global pharmaceutical company Pfizer to advance Repligen’s spinal muscular atrophy (SMA) program, which includes RG3039, an experimental drug for SMA whose development MDA has supported.