Cure SMA - Connecticut Chapter

Directions Website Contact the organization

Cure SMA - Connecticut Chapter Cure SMA CT Chapter

Incredible news for the SMA community!
09/11/2026

Incredible news for the SMA community!

Incredible news for the SMA community! Scholar Rock has received FDA approval for ISEMBYLD™ (apitegromab-mstn) to treat spinal muscular atrophy (SMA) in adults and children 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. This approval brings the first muscle-targeted treatment to the SMA community.

This is a meaningful step forward in our collective mission to address the critical unmet needs of individuals living with SMA while empowering families with more options for care.

Learn more about this exciting development and what it means for individuals and families impacted by SMA: https://www.curesma.org/scholar-rock-receives-approval-for-isembyld-for-the-treatment-of-sma/

More treatment progress!
04/06/2026

More treatment progress!

Scholar Rock has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration for apitegromab, an investigational therapy for spinal muscular atrophy.

Scholar Rock anticipates FDA acceptance of the BLA within 30 days of resubmission and a review period of up to six months from the date of resubmission.

Cure SMA will continue to monitor progress and share updates with the community.

Click the link to read the full update: https://www.curesma.org/scholar-rock-resubmits-biologics-license-application-bla-for-apitegromab-to-the-fda/

Incredible news for the SMA Community!
03/30/2026

Incredible news for the SMA Community!

Biogen today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for the High Dose regimen of SPINRAZA to treat people living with SMA. With broad label approval for all those living with SMA, this is an excellent result for the SMA community.

This FDA approval marks another important step forward in the ongoing effort to treat and manage SMA. The High Dose regimen of SPINRAZA has the potential to provide additional benefit for those currently receiving treatment, offering new possibilities for improved outcomes and quality of life. Cure SMA is committed to working closely with Biogen, healthcare providers, and the SMA community to support timely education and access to this new dosing option, in alignment with its approved use.

To learn more, please visit: https://www.curesma.org/biogen-receives-fda-approval-of-high-dose-spinraza-for-the-treatment-of-sma/

Address

Hartford, CT

Website

Alerts

Be the first to know and let us send you an email when Cure SMA - Connecticut Chapter posts news and promotions. Your email address will not be used for any other purpose, and you can unsubscribe at any time.

Contact The Organization

Send a message to Cure SMA - Connecticut Chapter:

Shortcuts

Share

Category