SMA2d
Raising SMN protein levels may be helpful early step in SMA treatment
Combining a premade SMN protein with a Spinraza-like drug (targets SMN2 gene) helped patient-derived motor neurons; potential ...
SMA4d
Zolgensma into spinal canal drives SMA type 2 motor gains
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 ...