The term Spinal Muscular Atrophy (SMA) identifies a group of genetic disorders affecting spinal motor neurons. It is caused by the loss of the SMN1 gene, resulting in degeneration of spinal alpha motor neurons and muscle atrophy. This study is focused on innovative gene therapies with onasemnogene abeparvovec approved in Italy in March 2021 with full reimbursement by the National Health Service.
View Article and Find Full Text PDFOrphanet J Rare Dis
October 2020