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          Spinal Muscular Atrophy

          This shows baby Arabella Smygov following gene therapy treatment for SMA
          FeaturedGeneticsNeurologyNeuroscience
          ·June 10, 2019·7 min read

          ‘Like Looking at a Miracle’: Baby Blossoms Thanks to Gene Therapy For Spinal Muscular Atrophy

          Zolgensma, an FDA approved gene therapy, supplements the production of the SMN protein to improve motor neuron function in children with spinal muscular atrophy after just one dose.
          Read More
          neurons
          FeaturedNeuroscienceOpen Neuroscience Articles
          ·November 2, 2017·3 min read

          Synaptic Disorder

          Targeting autophagy may help in the battle against ALS and other diseases that affect motor neurons, researchers report.
          Read More
          motor neurons
          FeaturedGeneticsNeurology
          ·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.
          Read More
          Image shows a DNA strand.
          FeaturedGenetics
          ·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.
          Read More
          Image shows the SMN protein in motor neurons.
          FeaturedGeneticsNeurologyOpen 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.
          Read More
          Image shows neurons.
          FeaturedGeneticsNeurologyOpen 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.
          Read More
          Image shows the spine.
          FeaturedNeurology
          ·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.
          Read More
          Image shows C. elegans.
          FeaturedGenetics
          ·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.
          Read More
          Image shows baby feet.
          FeaturedNeurology
          ·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.
          Read More
          Image shows SMA motor neurons.
          FeaturedGeneticsNeurology
          ·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.
          Read More
          FeaturedNeurology
          ·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.
          Read More
          An image of fly spinal cord equivalent is shown.
          FeaturedNeuroscience
          ·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.
          Read More
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          Facial Expressions Reveal Hidden Cognitive States

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          Chronic Pain Linked to Neuron Overactivity in the Brainstem

          This shows DNA and a child's head.

          New Autism Data Reveals Insights Into Profound Cases

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          Wellbeing May Help Protect Memory in Middle Age

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