Newsstand Menu

CSHL Stories and Media archive for Tag: SMA

FDA approval of life-saving SMA drug is hailed by its researcher-inventor at CSHL

12/24/2016

Nusinersen (Spinraza) will be used to treat young children with a severe and potentially fatal illness called spinal muscular atrophy (SMA). Read the story »

One experiment: A drug for SMA visibly repairs defective cells

12/1/2016

Professor Adrian Krainer develops a treatment for spinal muscular atrophy, the leading genetic cause of infant death. Read the story »

Emma’s Story: With science on her side, one little girl finds hope

11/22/2016

Emma's prognosis was grim, SMA. That's now changing, thanks to the tireless work of Dr. Adrian Krainer and his lab. Watch the video »

Just what is RNA splicing?

10/17/2016

Youreka Science and Cold Spring Harbor Laboratory team up to explain how RNA splicing happens. Watch the video »

Base Pairs Episode 5: “Sink or swim”

10/15/2016

When Adrian Krainer was just 27 years old, he was offered the opportunity to run his own lab. Now, research from his lab has contributed significantly Listen to the podcast »

“Sink or swim”

10/15/2016

This episode of Base Pairs follows Professor Adrian Krainer and his experience as the first CSHL fellow. Read the story »

Candidate drug for spinal muscular atrophy, with roots in CSHL research, passes major hurdle

8/4/2016

Biotechnology firms Biogen and Ionis Pharmaceuticals cut short a phase-3 clinical trial for the investigational drug nusinersen as it met endpoint. Read the story »

Adrian Krainer of Cold Spring Harbor Laboratory elected to American Academy of Arts and Sciences

4/20/2016

Adrian Krainer was elected to the American Academy of Arts and Sciences. Read the story »

Mechanistic insights into spinal muscular atrophy suggest new paths for treatment

1/12/2015

Researchers find that in mouse models of the disease subcutaneous drug administration is sufficient to treat SMA Read the story »

First animal model of adult-onset SMA sheds light on disease progression and treatment

9/9/2013

Another proof of principle for TSUNAMI method in modeling spinal muscular atrophy and other splicing-related illnesses Read the story »

CSHL-led team introduces new method to closely model diseases caused by splicing defects

8/15/2012

Researchers find novel method of making animal models for human genetic disease research. Read the story »

A new drug for spinal muscular atrophy (SMA)

3/29/2012

CSHL Professor Adrian Krainer has developed a new drug for spinal muscular atrophy (SMA). Watch the video »

Splicing studies detail a protein’s role in creating cancer’s mixed messages

2/13/2012

Adrian Krainer discusses alternative splicing and how it can be used to treat diseases like cancer and spinal muscular atrophy. Read the story »

Antisense therapy delivers long-term correction of severe spinal muscular atrophy in mice

10/5/2011

Researchers report surprising results that suggest that spinal muscular atrophy might not exclusively affect the motor neurons in the spinal cord. Read the story »

Collaboration column: SMA therapeutics and potential drug target for schizophrenia

3/11/2011

CSHL's Adrian Krainer works on treatment for spinal muscular atrophy that reverses the disease's symptoms. Read the story »

Cookie	Duration	Description
cookielawinfo-checkbox-analytics	11 months	This cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Analytics".
cookielawinfo-checkbox-functional	11 months	The cookie is set by GDPR cookie consent to record the user consent for the cookies in the category "Functional".
cookielawinfo-checkbox-necessary	11 months	This cookie is set by GDPR Cookie Consent plugin. The cookies is used to store the user consent for the cookies in the category "Necessary".
cookielawinfo-checkbox-others	11 months	This cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Other.
cookielawinfo-checkbox-performance	11 months	This cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Performance".
viewed_cookie_policy	11 months	The cookie is set by the GDPR Cookie Consent plugin and is used to store whether or not user has consented to the use of cookies. It does not store any personal data.