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Phase II/III, open-label study evaluating risdiplam in infants 1 to 7 months of age who had type 1 spinal muscular atrophy, which is characterized by the infant not attaining the ability to sit without support.
Primary outcomes measures:
Safety, pharmacokinetics, pharmacodynamics (including the blood SMN protein concentration), and the selection of the risdiplam dose for part 2 of the study.
The authors' conclusion was that in infants with type 1 spinal muscular atrophy, treatment with oral risdiplam led to an increased expression of functional SMN protein in the blood.
References:
Baranello et al. 2021 N Engl J Med 384: 915-923
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