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.
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.
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.
Krainer awarded Watanabe Prize in Translational Research
June 16, 2022
Indiana University School of Medicine honored Krainer for his pioneering work on RNA splicing, which led to the first FDA-approved SMA therapeutic.
The rise of RNA therapeutics
October 14, 2021
RNA has been making waves as a new approach to prevent or treat diseases, including COVID-19 and spinal muscular atrophy.
Krainer wins Gabbay Award for SMA research
June 28, 2021
CSHL Professor Adrian Krainer won the Jacob and Louise Gabbay Award in Biotechnology and Medicine for his work on spinal muscular atrophy (SMA).
Envisagenics and Biogen partner for RNA splicing research
June 9, 2021
CSHL spin-out company Envisagenics teams up with Biogen to advance research in RNA-based therapeutics for central nervous system diseases.
Krainer wins 2021 Wolf Prize in Medicine
February 17, 2021
CSHL Professor Adrian Krainer was awarded the 2021 Wolf Prize in Medicine for his work on RNA splicing.
CSHL tops Bush Center’s Innovation Impact Productivity Score
July 13, 2020
Cold Spring Harbor Laboratory ranks #1 in Innovation Impact Productivity Score among Pure Research/Health Care Institutions by the Bush Center.