https://journals.viamedica.pl/neurologia_neurochirurgia_polska/article/view/64309
Spinal muscular atrophy — new therapies, new challenges
Vol 54, No 1 (2020)
Invited review article
Published online: 2020-01-10
Submitted: 2019-05-09
Accepted: 2019-10-19
RG7916 and LMI070 or SMN1 gene replacement therapy (AAV9-SMN).