News & Updates

July 2026 — ALS: a licensing agreement to develop a new therapy from our miR-129 patent

The Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico has signed an exclusive licensing agreement with Life BioCeed, an international venture capital firm, to develop miR-129 inhibitors for amyotrophic lateral sclerosis. The approach, protected by a patent family of the Policlinico, was developed by our group in collaboration with the University of Milano-Bicocca and improved neurological function in preclinical models. Read more →

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July 2026 — Human 3D organoids for motor neuron diseases: our review in Nature Reviews Neuroscience

Frizzi, Barbosa Correia, Faravelli and colleagues review how human brain and spinal cord organoids are providing mechanistic insight into ALS, SMA and other motor neuron diseases, and how they can accelerate therapy development. Read more →

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June 2026 — SOD1 variants are more common than expected: a UK Biobank study for early intervention in ALS

In a study published in Annals of Neurology with University College London, we analysed the genetic data of about 470,000 people and identified over 100 presymptomatic carriers of pathogenic SOD1 variants. Since targeted therapies for SOD1-ALS exist, identifying carriers early opens the way to timely, personalised treatment. Read more →

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February 2026 — SMA begins during embryonic development: our study in Nature Communications

Using spinal and cerebral organoids derived from patients, with Humanitas University and Columbia University, we showed that SMN deficiency alters neural progenitors long before symptoms appear, and that early antisense oligonucleotide treatment rescues these developmental alterations: strong support for newborn screening and presymptomatic treatment. Read more →

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September 2025 — Rare SMN1 variants identified in people with SMA

In Neurology Genetics we describe single-nucleotide SMN1 variants and hybrid SMN1/SMN2 genes in a 20-year cohort of 149 patients: rare variants that standard screening can miss, with direct consequences for diagnosis and access to therapies. Read more →