Spinal Muscular Atrophy (SMA)

MDA Scientific Conference To Emphasize Therapy Development

The Muscular Dystrophy Association’s annual conference being held in Washington, D.C., on April 21-24, 2013, is centered on the theme Therapy Development for Neuromuscular Diseases: Translating Hope into Promise.

SMA Research Briefs: Testing Drugs from Other Diseases

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

SMA Antisense Drug Shows Safety, Tolerability in Phase 1 Trial

An experimental drug designed to treat the underlying molecular defect in spinal muscular atrophy (SMA) has shown encouraging results in a phase 1 trial.

SMA Research Briefs: Gene Therapy, New SMA Mouse

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.   

New Guidelines on Genetic Testing in Children

As scientists learn more about what our DNA can tell us about health and disease, public interest has intensified and genetic testing has become increasingly common. In response, the American Academy of Pediatrics (AAP) and the American College of Medical Genetics and Genomics (ACMG) have released new guidelines to address updated technologies and new uses of genetic testing and screening in children.

SMA 'NeuroNEXT' Biomarkers Study Seeks Participants

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.  

$13.6 Million in New MDA Grants Promote Understanding, Treatment of Neuromuscular Diseases

The Muscular Dystrophy Association has awarded 44 new grants totaling $13.6 million to advance the understanding and treatment of neuromuscular diseases. The new grants, most of which took effect Feb. 1, encompass a range of diseases covered by MDA’s research program, and they support innovative approaches to basic research and new drug development.

In addition to addressing 16 specific neuromuscular diseases under MDA’s umbrella, the grants also fund research into muscular dystrophy in general, and research into muscle physiology related to neuromuscular disease.

SMA — John Manfredi, Ph.D.

John Manfredi, chief scientific officer at Sfida BioLogic in Salt Lake City, Utah, was awarded an MDA research grant totaling $161,995 over a period of two years to study the potential of new compounds for treatment of spinal muscular atrophy (SMA). The new grant complements previous MDA-funded research by Manfredi into potential therapeutics for SMA.

MDA Webinar Discusses Spinal Bracing and Surgery in Children

A one-hour, MDA-sponsored webinar features two physicians and the parent of a child with spinal muscular atrophy (SMA) who has undergone bracing and surgery for a spinal curvature, as well as questions and answers from listeners.

SMA — Christine DiDonato, Ph.D.

Christine DiDonato, assistant professor of pediatrics at Northwestern University in Chicago, Ill., was awarded an MDA research grant totaling $405,000 over a period of three years to test treatment strategies for spinal muscular atrophy (SMA).

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