FDA Approves First And Only Innovative “Gene Therapy” For Children With Spinal Muscular Atrophy (SMA)

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By Gaurav Bedse, Ph.D, Research Instructor, Vanderbilt University Medical Center, Nashville, TN May 26, 2019

The U.S. Food and Drug Administration (FDA) approved the gene therapy “Zolgensma” to treat children less than 2 years of suffering from spinal muscular atrophy (SMA). Zolgensma is the first and only gene therapy approved by FDA for the treatment of SMA. Zolgensma is designed to address the route cause of the disease and it will be one time therapy. It will be the most expensive therapy so far costing $2.1 million.

SMA is a debilitating genetic disease affecting the parts of the nervous system that controls voluntary muscle movements. SMA affects 1 in 6,000 to 1 in 10,000 children. SMA involves loss of motor neurons in the spinal cord.  The nervous cells that are involved in muscle movements are called as motor neurons. A gene, which is critical for normal functioning of the motor neurons, is missing from the motor neurons in SMA children. The muscles affected in the SMA are the muscles near to the center of the body such as back, shoulders, hips and thighs. Children with SMA have problem in holding their head up, swallowing and breathing. These symptoms are present at birth or may present by the age of 6 months. Most of the children do not survive past early childhood due to respiratory failure.

Zolgensma will be magical therapy for the children suffering from SMA. It is adeno-associated virus vector based therapy that target route cause of the disease. One time administration of Zolgensma will deliver the gene that is missing from SMA children and will restore the normal functioning of the motor neurons. Zolgensma could create a lifetime of possibilities for the children and families impacted by this devastating condition. Zolgensma will be made available in the US and will be marketed by AveXis, a Novartis company.


  1. https://www.novartis.com/news/media-releases/avexis-receives-fda-approval-zolgensma-first-and-only-gene-therapy-pediatric-patients-spinal-muscular-atrophy-sma
  2. https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease

Image: Illustration purpose only, credited to Shivali

Let’s meet the author:

Gaurav Bedse, Ph.D,

Research Instructor, Vanderbilt University Medical Center, Nashville, TN

He is a neuroscientist with a solid background in preclinical programs, drug discovery, and translational sciences who has a passion for applying basic sciences to advancing medicines to treat neurological diseases.

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