Combining a premade SMN protein with a Spinraza-like drug (targets SMN2 gene) helped patient-derived motor neurons; potential ...
The Phase 3 STEER trial enrolled more than 100 children with SMA type 2, ages 2-17, who were able to sit, but hadn't walked ...
The top 10 most-read stories published by SMA News Today in 2024 focused on symptoms, treatments, and diagnosis.
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