1. C2.4 Historic breakthrough in the treatment of spinal muscular atrophy (5q-sma): nusinersen (spinraza) is the first drug approved for sma. (4th October 2017) Authors: Scoto, M; Main, M; Munot, P; Manzur, A; Muntoni, F Journal: Archives of disease in childhood Issue: Volume 102(2017)Supplement 3 Page Start: A12 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. G299 Nusinersen (spinraza) is the first drug approved for spinal muscular atrophy (sma): initial experience in patients with sma type 1 treated in the expanded access program (eap). (12th March 2018) Authors: Scoto, M; Main, M; Munot, P; Edel, L; Abel, F; Chan, E; Manzur, A; Muntoni, F Journal: Archives of disease in childhood Issue: Volume 103(2018)Supplement 1 Page Start: A122 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗