Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience

Affiliations

• ¹ Department of Neurosciences, Psychiatry and Anaesthesiology and Centro Clinico Nemo, Messina, Italy.
• ² Paediatric Neurology and Centro Clinico Nemo, Catholic University and Policlinico Gemelli, Rome, Italy.
• ³ Centro Clinico Nemo, Niguarda Hospital, Milano, Italy.
• ⁴ Neuromuscular Disease Unit, G. Gaslini Institute, Genoa, Italy.
• ⁵ Department of Laboratory Medicine, Unit of Molecular Medicine, Bambino Gesù Hospital, Genoa, Italy.
• ⁶ Department of Laboratory Medicine, Unit of Molecular Medicine, Bambino Gesù Hospital, Rome, Italy.
• ⁷ Associazione Famiglie SMA, Italy.
• ⁸ Paediatric Neurology and Centro Clinico Nemo, Catholic University and Policlinico Gemelli, Rome, Italy. Electronic address: eugeniomaria.mercuri@unicatt.it.