Access to treatment is an issue for patients across the world, especially for those with rare diseases. Laurie Young, who was diagnosed with type 2 spinal muscular atrophy (SMA) at…
Genentech has recently released the data from its SUNFISH trial, a study of the effects of risdiplam on spinal muscular atrophy (SMA) patients. Preliminary data from the JEWELFISH trial has…
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