Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study
Audic F, de la Banda MGG, Bernoux D, Ramirez-Garcia P, Durigneux J, Barnerias C, Isapof A, Cuisset JM, Cances C, Richelme C, Vuillerot C, Laugel V, Ropars J, Altuzarra C, Espil-Taris C, Walther-Louvier U, Sabouraud P, Chouchane M, Vanhulle C, Trommsdorff V, Pervillé A, Testard H, Lagrue E, Sarret C, Avice AL, Beze-Beyrie P, Pauly V, Quijano-Roy S, Chabrol B, Desguerre I
Orphanet J Rare Dis. 2020. 12;15(1):148.
This study reports data retrospectively collected from 23 French centers for neuromuscular diseases on 204 patients with Spinal Muscular Atrophy (type 1 or 2) treated by Nusinersen.
Patients treated over 2 years showed significantly higher MFM total scores after 1 year, indicating that their motor function had improved. The scores were higher in the axial and proximal motor function (D2) and distal motor function (D3), but there was no significant difference for standing and transfer scores (D1).
Keywords: MFM, Nusinersen, Spinal Muscular Atrophy type I et II
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