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
Corti lab is focused on cellular (stem cells) and molecular therapies (gene therapies, antisense oligonucleotides) for neurodegenerative diseases in particular motor neuron diseases (Amyotrophic Lateral Sclerosis, ALS, Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy with Respiratory Distress type 1, SMARD1), neuropathies (CMT2A) and neuromuscular diseases.
We established patient-specific 2D and 3D models (iPSC-derived organoids patterned into brain/spinal cord) to study disease pathogenesis and develop novel therapeutic strategies for neurological diseases.
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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atrophy, with approval for use in patients with type 2 and type 3 spinal muscular atrophy granted on the basis of unpublished data.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.