New Research In Treatment for Spinal Muscle Atrophy (SMA)

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Science Translation Medicine has just published groundbreaking research on spinal muscle atrophy (SMA.)

SMA is a set of inherited muscle diseases that cause muscle to continuously degenerate and weaken. The disease requires extensive and ongoing medical care. The disease process usually ends with death. In fact, SMA is one of the most common genetic causes of death in childhood. Sadly, there is no known cure or effective treatment.

The research by University of Sheffield demonstrated a novel gene transfer system that has the promise of efficient therapeutic treatment for SMA patients. I will let you read the research process below, but the approach has the possibility of establishing gene therapy with the use of just a single injection. 

It is important to note that test was conducted using animals, but these findings undoubtedly put science that much closer to actual gene therapy treatments for SMA patients.

Research in depth:

New therapy offers hope to Spinal Muscular Atrophy (SMA) patients

Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy

New Novel Gene Treatment For Spinal Muscular Atrophy

To donate to SMA: 

Spinal Muscular Atrophy Foundation

 To support legislative action:

Legislation to enhance federal support for Spinal Muscular Atrophy (SMA) research, The SMA Treatment Acceleration Act