SMA Angels Charity, Inc.

04/02/2026

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/

03/24/2026

Cure SMA is excited to announce the main events held at this year’s Annual SMA Conference:

Meet & Greet
Family Friendly Researcher Poster Session
PJ Party & Movie Night
…and many more socials and networking events throughout the weekend!

Registration will be closing on Friday, May 8 – If you have not registered yet, be sure to register and book your hotel room today!

Please contact [email protected] with any questions.

https://www.accelevents.com/e/2026-annual-sma-conference

03/24/2026

Get the scoop: https://bit.ly/3MUkprW
Understanding the genetics behind Spinal Muscular Atrophy can help explain why symptoms develop and why the condition can vary from person to person. Mutations in the SMN1 gene reduce the amount of SMN protein needed for healthy motor neurons, while the SMN2 gene can produce small amounts of this protein and influence disease severity.

Accurate, early diagnosis is critical—helping individuals access treatment sooner and supporting better long-term outcomes. 🧬

03/24/2026

Get to know the different SMA types and connect with shared experiences: https://bit.ly/41AE3gr

03/24/2026

Recognize SMA symptoms early and support others on the same journey: https://bit.ly/3UogDan

02/19/2025

The new tablet formulation, which can be swallowed whole or dissolved in water, is expected to offer more convenient dosing for patients. https://bit.ly/412ylTF

02/15/2025

The National Institutes of Health (NIH) recently announced a major reduction in grant funding that would have a catastrophic impact on research, especially in the neuromuscular disease field. NIH funding has led to amazing discoveries, including the underlying mechanisms of genetic neuromuscular diseases, breakthroughs in gene therapy for neuromuscular diseases, and effective treatments for spinal muscular atrophy, Pompe disease, Duchenne muscular dystrophy, and more.

Cutting funding now would stop all this remarkable progress in its tracks.

The National Institutes of Health (NIH) has implemented a harmful 15% cap on indirect costs for research grants, threatening future progress in neuromuscular disease research. Indirect costs are essential to keeping labs running, supporting clinical trials, and ensuring that progress is made toward new treatments.

40 neuromuscular advocacy organizations have united to demand the NIH immediately reverse this policy, which could catastrophically harm neuromuscular disease research, drug development, and care ecosystem. Specifically, this policy would halt research, shut down labs, and stall future medical breakthroughs that are important to the MDA community.

Read the statement here:https://d3dkdvqff0zqx.cloudfront.net/groups/mda/attachments/2(1).13.2025%20Neuromuscular%20Advocacy%20Groups%20Ask%20NIH%20To%20Retract%2015%20Percent%20Indirect%20Cost%20Cap.pdf

We need to act now to protect the decades of progress we have made in neuromuscular disease research. TAKE ACTION and contact your representative to oppose NIH cuts: https://www.votervoice.net/MDA/Campaigns/121428/Respond

02/15/2025

FDA approves Roche’s Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA)

New tablet form of Evyrsdi to become available soon!

https://www.roche.com/media/releases/med-cor-2025-02-12?fbclid=IwZXh0bgNhZW0CMTEAAR3BZXUu65vkzk8Wj0JzQ1fmVBASU-FV5YS4uG0Llh0rvOHZW5Fygz6ajO8_aem_sUzZev3WIjqnGrH6hEK5KA

02/15/2025

How do the main types of SMA differ from one another? We break down the characteristics here: https://bit.ly/3ZY6GTm

02/15/2025

Also known as Werdnig-Hoffmann disease, this infantile-onset form accounts for roughly 60% of cases. Take a look at its characteristics here: https://bit.ly/4ge67vJ

02/15/2025

Explore approved SMA DMTs, how they're administered, and how they work in this treatment-focused resource: https://bit.ly/4eNoCp5