New therapies and systemic impact of Spinal Muscular Atrophy

Our team from the Dino Ferrari Center of the University of Milan discusses the advancements and challenges of new therapies for patients with Spinal Muscular Atrophy in Nature Reviews in Neurology.

The prestigious journal “Nature Reviews Neurology” has invited Prof. Stefania Corti, with the contribution of Dr. Irene Faravelli, to comment on two recent scientific studies regarding the recent, important progresses in spinal muscular atrophy (SMA) therapeutic approaches. The approval of three innovative therapies (gene therapy, oligonucleotides and small molecules), which are available to patients of the Policlinico di Milano, has in fact determined the advent of a new era for SMA treatment.

The article discusses the latest scientific evidence regarding the extra-neural involvement of SMA and illustrates how the systemic aspects of the disease can be addressed by the new therapies (https://doi.org/10.1038/s41582-020-00411-6). This prestigious recognition goes to the team of Prof. Corti, who for years has been involved in basic scientific research and clinical trials, aimed at investigating the causes and identifying new treatments for SMA and other motor neuron diseases.

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