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Spinal Muscular Atrophy

3D medical illustration of a human brain showing distinct color-coded anterior and posterior developmental regions converging within a single skull. Caption reads "The Human Brain Is Two Distinct Organs Packaged Together."

Featured Genetics Neuroscience

·September 18, 2026·6 min read

The Brain Is Two Separate Organs Joined by Evolution

A new study reveals the human brain is composed of two independently evolved organs, enabling the first lab-grown human hindbrain neurons.

This shows baby Arabella Smygov following gene therapy treatment for SMA

Featured Genetics Neurology Neuroscience

·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.

neurons

Featured Neuroscience Open 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.

motor neurons

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.

Image shows a DNA strand.

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.

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.

Image shows neurons.

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 spine.

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.

Image shows C. elegans.

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.

Image shows baby feet.

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.

Image shows SMA motor neurons.

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

·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.

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