Barriers in the access to expensive genetic therapies for spinal muscular atrophy
Although the inherited neuromuscular disease spinal muscular atrophy is considered a rare disease, it has been on the forefront of genetic therapies research, with three therapies receiving FDA approval since 2016. Spinal muscular atrophy is an autosomal recessive loss of function disease that results progressive degeneration of the motor neurons which leads to continued progressive […]