Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3

2020189 citationsJournal Articlebronze Open Access

Authors

Lorenzo Maggi · Fondazione IRCCS Istituto Neurologico Carlo Besta

Luca Bello · University of Padua

Silvia Bonanno · Fondazione IRCCS Istituto Neurologico Carlo Besta

Alessandra Govoni · Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Claudia Caponnetto · Ospedale Policlinico San Martino

Luigia Passamano · University of Campania "Luigi Vanvitelli"

Marina Grandis

Abstract

Our data provide further evidence of nusinersen safety and efficacy in adult SMA2 and SMA3, with the latter appearing to be cumulative over time. In patients with extremely advanced disease, effects on residual motor function are less clear.

Topics & Keywords

Neurogenetic and Muscular Disorders Research RNA modifications and cancer Congenital Anomalies and Fetal Surgery

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Journal of Neurology Neurosurgery & Psychiatry

Volume 91, Issue 11, pp. 1166-1174

DOI: 10.1136/jnnp-2020-323822

Field-Weighted Citation Impact: 12.59