The SMA Trust: the role of a disease-focused research charity in developing treatments for SMA

Christie-Brown, V; Mitchell, J; Talbot, K

Christie-Brown, V (reprint author), SMA Trust, 3 Wychwood Court, Moreton In Marsh GL56 0JQ, Glos, England.

GENE THERAPY, 2017; 24 (9): 544

Abstract

SMA is a rare hereditary neuromuscular disease that causes weakness and muscle wasting as a result of the loss of spinal motor neurons. In its most se......

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