Investigating the therapeutic potential of the glucocorticoid antagonist, mifepristone, for the treatment of spinal muscular atrophy
(2023)
Thesis
Sutton, E. R. (2023). Investigating the therapeutic potential of the glucocorticoid antagonist, mifepristone, for the treatment of spinal muscular atrophy. (Thesis). Keele University
Spinal muscular atrophy (SMA) is a neuromuscular disease characterised by loss of alpha motor neurons and muscle atrophy. When untreated, mortality occurs within the first two years of life in the most severe cases. Extensive research has led to thre... Read More about Investigating the therapeutic potential of the glucocorticoid antagonist, mifepristone, for the treatment of spinal muscular atrophy.