Genetics

Candidate drug provides benefit in SMA animal models

In a new publication that appears in Human Molecular Genetics, the laboratory of Christine DiDonato, PhD reports on their pharmacological characterization of the drug RG3039, demonstrating that it can extend survival and ...

Neuroscience

Study offers new approach for spinal muscular atrophy

Spinal muscular atrophy is a debilitating neuromuscular disease that in its most severe form is the leading genetic cause of infant death. By experimenting with an ALS drug in two very different animal models, researchers ...

Surgery

High mortality, morbidity with early-onset scoliosis surgery

(HealthDay)—Surgery for patients with early-onset scoliosis is associated with an 18 percent mortality rate and an 84 percent complication rate, according to research published in the Feb. 15 issue of Spine.

Medical research

Mutated gene causes nerve cell death

Researchers identify new mechanism in the onset of incurable nerve disease The British astrophysicist Stephen Hawking is likely to be the world's most famous person living with amyotrophic lateral sclerosis (ALS), also known ...

Neuroscience

Researcher advancing motor neuron studies

A University of Connecticut researcher is advancing the understanding of the devastating inherited condition known as spinal muscular atrophy.

Medical research

New research sheds light on childhood neuromuscular disease

A study by scientists at the Motor Neuron Center at Columbia University Medical Center suggests that spinal muscular atrophy (SMA), a genetic neuromuscular disease in infants and children, results primarily from problems ...

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