Nusinersen for SMA: expanded access programme

Farrar, MA; Teoh, HL; Carey, KA; Cairns, A; Forbes, R; Herbert, K; Holland, S; Jones, KJ; Menezes, MP; Morrison, M; Munro, K; Villano, D; Webster, R; Woodcock, IR; Yiu, EM; Sampaio, H; Ryan, MM

Farrar, MA (reprint author), Sch Womens & Childrens Hlth, High St, Randwick, NSW 2031, Australia.; Farrar, MA (reprint author), Sydney Childrens Hosp, High St, Randwick, NSW 2031, Australia.

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 2018; 89 (9): 937

Abstract

Background Spinal muscular atrophy (SMA) is a devastating motor neuron disorder causing progressive muscle weakness and respiratory insufficiency. We ......

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