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Spinal Muscular Atrophy

·July 16, 2012·2 min read

Gene Therapy Treatment Extends Lives of Mice with Fatal Disease, Spinal Muscular Atrophy

Mice born with Spinal Muscular Atrophy typically only live five to six days. University of Missouri researchers introduced the SMN gene into the mice’s central nervous systems and were able to extend their lives 10-25 days longer.

An image of fly spinal cord equivalent is shown.

Featured Neuroscience

·October 11, 2012·6 min read

Novel Mechanisms Underlying Major Childhood Neuromuscular Disease Identified

A study suggests that spinal muscular atrophy (SMA), a genetic neuromuscular disease in infants and children, results primarily from motor circuit dysfunction, not motor neuron or muscle cell dysfunction, as is commonly thought. In a second study, the researchers identified the molecular pathway in SMA that leads to problems with motor function.

Featured Neurology

·December 18, 2013·3 min read

Researchers Provide New Insights into Cause of Human Neurodegenerative Disease

Researchers identify Neurexin2 as a new target for potential treatment of spinal muscular atrophy.

Featured Genetics Neurology

·September 24, 2015·5 min read

Surprising Similarities Between Childhood Disorder and ALS

A new study uncovers molecular changes which may explain why motor neurons, rather than other neurons, are affected by the childhood illness, SMA.

Featured Neurology

·November 9, 2015·3 min read

A Breakthrough in Understanding Childhood Motor Neuron Disease

Researchers report insufficient blood supply could contribute to motor neuron loss in SMA.

Featured Genetics

·July 11, 2016·5 min read

Protein Deficiency May Cause Spinal Muscular Atrophy

A new study implicates a key cellular mechanism as defective in SMA for the first time, providing a new lead for developing future interventions.

Featured Neurology

·July 25, 2016·4 min read

New Drug Could Help Treat Spinal Muscular Atrophy

A new drug shows promise for improving outcomes for people with SMA.

Featured Genetics Neurology Open Neuroscience Articles

·September 20, 2016·4 min read

Trial Offers Hope of a Treatment for Spinal Muscular Atrophy

Researchers believe they have discovered a promising new treatment for spinal muscular atrophy, a leading genetic cause of death in children.

Image shows the SMN protein in motor neurons.

Featured Genetics Neurology Open Neuroscience Articles

·April 17, 2017·5 min read

Common Drug Could Help Treat ALS and SMA

Researchers have identified a compound that can stabilize a protein implicated in ALS and SMN.

Featured Genetics

·May 15, 2017·4 min read

New Gene Identified in ALS

Researchers implicate a variant in the UBQLN4 gene as a possible cause of ALS. The variant disrupts cellular processes that drive motor neuron development.

Featured Genetics Neurology

·September 7, 2017·4 min read

Human Skin Cells Transformed Into Motor Neurons

WUSTL researchers have converted skin cells into motor neurons without going through the stem cell state. The new technique could help in the development of devastating neurodegenerative diseases, like ALS, that affect motor neurons.