Spinal Muscular Atrophy (SMA)
CSHL’s Adrian Krainer, Ph.D. has devoted his expertise in alternative splicing—a cellular process for editing RNA—towards fixing the genetic glitch that underlies Spinal Muscular Atrophy (SMA), a neuromuscular disease that’s currently the No.1 genetic cause of death among children under the age of two.
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SMA is a disorder affecting the motor neurons—nerve cells that control voluntary muscle movement. These cells are located in the spinal cord. Because the muscles cannot respond to signals from the nerves, they atrophy—weaken and shrink—from inactivity.
SMA can affect a child’s ability to crawl, walk, sit up, and control head movements. Severe SMA can damage the muscles used for breathing and swallowing.
Learn more about the story of a child’s illness, a scientist’s quest, and the circumstances that made possible the successful development of a life-saving treatment.
Can you ace this SMA quiz?
August 7, 2025
For Spinal Muscular Atrophy Awareness Month, test your knowledge of CSHL’s lifesaving SMA and RNA therapeutics research.
Giving hope to thousands of families
August 5, 2025
It’s SMA Awareness Month! See how CSHL’s fundamental biology research helped lead to the first successful treatment, and what's happened since.
Adrian Krainer wins 2025 Heinrich Wieland Prize
July 16, 2025
Krainer is recognized for his lifesaving research on mRNA splicing and RNA therapeutics.
Nature is trying to tell you something
February 3, 2025
Former Cold Spring Harbor Laboratory scientist Richard Roberts won the Nobel Prize in 1993. Here, he recalls the “failure” that made him famous.
Adrian Krainer wins Albany Prize for biomedical research
October 8, 2024
A pioneer in the burgeoning field of RNA therapeutics, Krainer has now received America’s second-highest prize in medicine.
President’s essay: The continuous cycle of discovery
May 30, 2024
CSHL President & CEO Bruce Stillman discusses our institution’s societal impacts and global connections as forces for further scientific progress.
Why some RNA drugs work better than others
March 6, 2024
CSHL’s Justin Kinney and Spinraza inventor Adrian Krainer tested the newly approved SMA treatment, risdiplam, and another RNA therapeutic, branaplam.
Adrian Krainer awarded honorary IADR membership
June 21, 2023
Krainer was recognized for his pioneering research on spinal muscular atrophy and RNA therapeutics.
Krainer named Society for RNA Therapeutics board member
April 18, 2023
He helped pioneer the field. It changed medicine. Now, he joins a group of renowned scientists and physicians aiming to take it to the next level.
The promising drug duo that may improve SMA treatment
July 11, 2022
Pairing Spinraza® with a second FDA-approved drug may be a new way to improve the drug’s therapeutic effects in spinal muscular atrophy patients.