We’re happy to share our next recipient of Cure SMA’s basic research funding, Michael Tellier, PhD, at the University of Leicester! Dr. Tellier was awarded $114,000 for his research project, “Characterization of the SMN-7SK complex in the regulation of SMN1 and SMN2 expression

This weekend, the President signed into law a government funding bill that included a provision in support of new SMA research at the National Institutes of Health (NIH).
 
Visit our news section to learn more:
curesma.org/gov-funding-bi…

Scholar Rock recently shared new data showing treatment with apitegromab was linked to improved muscle strength and range of motion, while being safe and well-tolerated in those with SMA types 2 and 3.
 
To learn more, visit out news section:
curesma.org/scholar-rock-n…

We’re happy to share our next recipient of Cure SMA’s basic research funding, Allison Ebert, PhD, at The Medical College of Wisconsin!
 
Read more about her project in our news section:
curesma.org/ebert-grant-an…

Yesterday, Biogen announced interim 6-month biomarker data from the initial 29 participants in the open-label Phase 4 RESPOND study.
 
To learn more, visit our news section:
curesma.org/biogen-present…

“Most Likely Not To…”, a first-of-its-kind musical comedy developed, written and performed by members of the SMA community, in collaboration with @Genentech.

Check it out on February 29, Rare Disease Day

mostlikelynotto.com

Melissa Bowerman, PhD, at Keele University was recently awarded $150,000 for her research project, “Defining the role of skeletal muscle in metabolic defects in SMA.”
 
Learn more about Dr. Bowerman and her research project:
curesma.org/bowerman-grant…

You did it!
 
We’ve now reached our goal of 100 percent newborn screening of spinal muscular atrophy (SMA) in all 50 states, with the addition of Hawaii at the start of 2024.

Thank you for everything you do to help improve the lives of everyone impacted by SMA.