Spinal Muscular Atrophy Insights

SMA baby achieves walking after early Evrysdi treatment

April 30, 2025 • Estimated Reading Time: 1 minute

Latest Research

Daghriri et al.

(2025) conducted a systematic review evaluating nusinersen's efficacy and safety in spinal muscular atrophy (SMA) types 1, 2, and 3.

The review synthesizes studies demonstrating improved motor function and milestone achievement across SMA subtypes, with a favorable safety profile characterized by manageable adverse events such as respiratory infections and reversible laboratory abnormalities.

The findings suggest nusinersen provides clinically meaningful benefits for SMA patients, supporting its role in symptom management.

The review contributes to treatment decision-making by clarifying the therapy's risk-benefit balance across different SMA types (Daghriri et al., 2025).

This analysis aids healthcare providers in optimizing therapeutic strategies for SMA populations.

Clinical Trials

This is a list of upcoming or ongoing clinical trials that are actively recruiting and have been listed or updated in the last two weeks:

A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Evaluating a New Knee-Ankle-Foot Brace to Improve Gait in Children With Movement Disorders

ALS/MND Natural History Study Data Repository

Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)

View the full list of clinical trials →

Community News

Spinal Muscular Atrophy UKApr 27, 2025

A huge GOOD LUCK to Morgan, Jamie, Mandeep, Todd, Zaf, and Tom who are taking on the London Marathon today in support of SMA UK! 🏃‍♂️💪

Together, they’ve already raised nearly £28,000! We’re beyond grateful for the time, energy, and passion they’ve poured into training and fundraising. Go smash it out there, we’re all cheering you on! 💙

Cure SMAApr 23, 2025

When Congress returns from its April recess, the U.S. House must identify $880 billion in healthcare-related savings that could impact how states deliver Medicaid and its caregiving services.

https://www.congress.gov/119/bills/hconres14/BILLS-119hconres14eas.pdf

Join Cure SMA in the coming weeks to share why Medicaid caregiving services are important to the SMA community.

Access to caregiving helps children and adults with SMA:

•Go to school

•Hold a job

•Live independently

•Participate in the community

📬 Tell Congress why Medicaid caregiving supports are essential.

https://www.votervoice.net/CureSMA/campaigns/110274/respond

SpinalMuscularAtrophy.netApr 28, 2025

"For many people, running to the store might feel like an inconvenience: a chore that interrupts their busy schedules. For me, though, it’s a full-day event that requires planning, patience, and plenty of assistance."