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The laboratory (CORTILAB) directed by Prof. Stefania Corti is one of the research facilities of the Dino Ferrari Center and the University of Milan (UNIMI) and is based in the Sforza and Invernizzi buildings at the Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan
Risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2)
Risdiplam is an oral small molecule approved for the treatment of patients with spinal muscular atrophy, with approval for use in patients with type 2 and type 3 spinal muscular atrophy granted on the basis of unpublished data. The drug modifies pre-mRNA splicing of the SMN2 gene to increase production of functional SMN. We aimed to investigate the safety and efficacy of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy.
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