SMA Archives | ��ϳԹ��Դ

Krainer included in Forbes’ Most Successful Immigrants list

Samuel Diamond — Mon, 15 Jun 2026 11:30:19 +0000

��ϳԹ��Դ (CSHL) Professor Adrian Krainer has been included in Forbes’ list of America’s Most Successful Immigrants. The magazine recognized Krainer for research that “led to the development of Spinraza, the first FDA-approved medicine for spinal muscular atrophy,” and for co-founding biotech company Stoke Therapeutics. After growing up in Uruguay, Krainer came to...

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“Philifant” sculpture honors research leading to Spinraza

Nick Wurm — Fri, 29 Aug 2025 14:40:15 +0000

On August 27, pioneering digital artist Phil L. Herold donated a chrome “Philifant” sculpture to ��ϳԹ��Դ (CSHL) for the Krainer lab’s transformative, life-saving research on spinal muscular atrophy (SMA). CSHL Professor Adrian Krainer’s work led to the development of Spinraza, the first FDA-approved treatment for SMA. At the time, the neurodegenerative disease...

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Can you ace this SMA quiz?

Communications Department — Thu, 07 Aug 2025 11:30:37 +0000

{“questions”:{“8r78v”:{“id”:”8r78v”,”mediaType”:”image”,”answerType”:”text”,”imageCredit”:””,”image”:”https:\/\/www.cshl.edu\/wp-content\/uploads\/2025\/07\/Richard_Roberts_Phillip_Sharp.jpg”,”imageId”:”74800″,”video”:””,”imagePlaceholder”:””,”imagePlaceholderId”:””,”title”:”In 1993, Richard Roberts and Phillip Sharp won the Nobel Prize in Physiology or Medicine for which revolutionary discovery?”,”desc”:”While CSHL played a role in all of these groundbreaking discoveries, only one is attributable to Roberts and Sharp. In 1977, CSHL\u2019s Richard Roberts and MIT\u2019s Phillip Sharp co-discovered split genes and RNA splicing while working with...

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��ϳԹ��Դ hope to thousands of families

Jen A. Miller — Tue, 05 Aug 2025 11:30:41 +0000

It’s a parent’s worst nightmare, finding out your newborn child may never walk or even stand on their own. That used to be the reality for as many as one in 6,000 babies born with spinal muscular atrophy (SMA), a genetic disorder that causes extreme nerve damage, muscle weakness, and sometimes, paralysis. For a long...

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Adrian Krainer wins 2025 Heinrich Wieland Prize

Nick Wurm — Wed, 16 Jul 2025 12:30:07 +0000

��ϳԹ��Դ Professor Adrian Krainer has won the 2025 Heinrich Wieland Prize. Awarded annually by the Boehringer Ingelheim Foundation, the €250,000 prize recognizes exceptional scientists whose pioneering research pushes the boundaries of knowledge in biochemistry, biology, chemistry, and physiology. Krainer received the award for his revolutionary RNA splicing research, which led to the...

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Nature is trying to tell you something

Jen A. Miller — Mon, 03 Feb 2025 12:30:52 +0000

“Nature is trying to tell you something.” Taken by itself, this quote from Nobel laureate and former ��ϳԹ��Դ (CSHL) scientist Richard Roberts suggests that the solutions to any number of problems may be within earshot. If only things were so simple. The fact is, scientific experiments go awry all the time. When...

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Adrian Krainer wins Albany Prize for biomedical research

Luis Sandoval — Tue, 08 Oct 2024 15:00:21 +0000

��ϳԹ��Դ (CSHL) Professor Adrian Krainer has won the Albany Prize, one of America’s largest awards for biomedical research. He now joins a prestigious list of recipients that includes names like Anthony Fauci, Emmanuelle Charpentier, and Jennifer Doudna. The Albany Prize recognizes scientists whose work has translated from “the bench to the bedside.”...

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President’s essay: The continuous cycle of discovery

Communications Department — Thu, 30 May 2024 11:30:43 +0000

��ϳԹ��Դ (CSHL) is a vibrant intellectual community that enables groundbreaking discovery. Our idyllic setting and state-of-the-art facilities offer a uniquely supportive environment for bioscience breakthroughs. However, CSHL’s community is not bound by the geography of our campus. Science worldwide has been strengthened by people who spend time on our campus and by...

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Why some RNA drugs work better than others

Jennifer Michalowski — Wed, 06 Mar 2024 12:30:20 +0000

Spinal muscular atrophy, or SMA, is the leading genetic cause of infant death. Less than a decade ago, ��ϳԹ��Դ (CSHL) Professor Adrian Krainer showed this brutal disease can be treated by tweaking a process called RNA splicing. This breakthrough resulted in Spinraza, the first effective treatment for SMA. It also opened a...

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Adrian Krainer awarded honorary IADR membership

Nick Wurm — Wed, 21 Jun 2023 23:00:43 +0000

��ϳԹ��Դ Professor Adrian Krainer has been named an honorary member of the International Association for Dental Research (IADR) for 2023. The IADR honored Krainer for fundamental and translational research that led to a life-saving treatment for spinal muscular atrophy (SMA). The group also acknowledged him for “enhancing our understanding of how [RNA]...

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Krainer named Society for RNA Therapeutics board member

Samuel Diamond — Tue, 18 Apr 2023 11:30:59 +0000

If RNA therapeutics were once considered the future of medicine, that future is now. Studies suggest RNA-based treatments have saved tens of millions of lives over the past few years. Now, the newly formed Society for RNA Therapeutics has come together to help further this cutting-edge science. On April 10, ��ϳԹ��Դ Professor...

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The promising drug duo that may improve SMA treatment

Luis Sandoval — Mon, 11 Jul 2022 11:30:15 +0000

In 2016, Spinraza® became a game-changer for spinal muscular atrophy (SMA) patients. It was the first FDA-approved treatment for the neurodegenerative disease, which is the leading genetic cause of infant death. The drug was conceived and developed by ��ϳԹ��Դ (CSHL) Professor Adrian Krainer and collaborators. But Krainer didn’t stop there. His lab...

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Krainer awarded Watanabe Prize in Translational Research

Nick Wurm — Thu, 16 Jun 2022 19:30:54 +0000

The Indiana University (IU) School of Medicine awarded ��ϳԹ��Դ Professor Adrian Krainer the August M. Watanabe Prize in Translational Research for his revolutionary work on a cellular process called RNA splicing. Krainer’s research led to the development of nusinersen (Spinraza®), the first FDA-approved treatment for spinal muscular atrophy (SMA). SMA is a...

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The rise of RNA therapeutics

Communications Department — Thu, 14 Oct 2021 11:30:27 +0000

« Previous Story Next Story » Most American newborns will arrive home from the hospital and start hitting their developmental milestones, to their parents’ delight. They will hold their heads up by about three months. They will sit up by six months. And they will walk by around their first birthday. But about 1 in...

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Krainer wins Gabbay Award for SMA research

Sara Roncero-Menendez — Mon, 28 Jun 2021 13:00:05 +0000

��ϳԹ��Դ Professor Adrian Krainer has been awarded the Jacob and Louise Gabbay Award in Biotechnology and Medicine for his role in the development of a therapy for the neuromuscular disease spinal muscular atrophy (SMA). Krainer shares the prize with C. Frank Bennett from Ionis Pharmaceuticals, with whom he collaborated to develop the...

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Envisagenics and Biogen partner for RNA splicing research

Luis Sandoval — Wed, 09 Jun 2021 12:00:04 +0000

The ��ϳԹ��Դ (CSHL) spin-out company Envisagenics and Biogen Inc. announced a new collaboration on May 13, 2021. This collaboration aims to advance RNA research in central nervous system diseases by leveraging the Envisagenics artificial intelligence–based platform called SpliceCore®. Genes, encoded in DNA, are transcribed into the related language of RNA. RNA is...

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Krainer wins 2021 Wolf Prize in Medicine

Sara Roncero-Menendez — Wed, 17 Feb 2021 17:30:36 +0000

��ϳԹ��Դ (CSHL) Professor Adrian Krainer has been awarded the 2021 Wolf Prize in Medicine. Krainer was chosen for his contributions in biochemistry and molecular genetics and his fundamental mechanistic discoveries on RNA splicing. He shares this honor with Dr. Joan Steitz of Yale University and Dr. Lynne Maquat of the University of...

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CSHL tops Bush Center’s Innovation Impact Productivity Score

Sara Roncero-Menendez — Mon, 13 Jul 2020 12:00:22 +0000

��ϳԹ��Դ (CSHL) is ranked #1 on Innovation Impact’s Productivity Score among Pure Research/Health Care Institutions, as well as #1 for Patent Citations by the George W. Bush Presidential Center. In their yearly report, “The Innovation Impact of U.S. Universities,” the Bush Center measures the innovations of American research institutions and highlights works...

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Krainer wins 2020 NYAS Innovators prize for SMA research

Sara Roncero-Menendez — Wed, 08 Jul 2020 12:00:19 +0000

��ϳԹ��Դ (CSHL) Professor Adrian Krainer has been named the 2020 Senior Scientist Winner of the Innovators in Science Award. Presented by the New York Academy of Sciences (NYAS) and Takeda Pharmaceuticals, the 2020 awards focus on research in rare diseases. Krainer was chosen for his pioneering research on the mechanisms and control...

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Adrian Krainer elected to the National Academy of Sciences

Sara Roncero-Menendez — Tue, 28 Apr 2020 17:34:31 +0000

Professor Adrian Krainer has been elected as a member of the National Academy of Sciences (NAS). He is one of 120 members and 26 international members chosen in 2020 in recognition of their distinguished and continuing achievements in original research. Krainer is best known for his work on RNA splicing, as well as his work...

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SMA Archives | ���ϳԹ���Դ