A new score combining compound muscle action potential (CMAP) amplitudes and motor score is predictive of motor outcome after AVXS-101 (Onasemnogene Abeparvovec) SMA therapy-Reference-Cited by-同舟云学术

A new score combining compound muscle action potential (CMAP) amplitudes and motor score is predictive of motor outcome after AVXS-101 (Onasemnogene Abeparvovec) SMA therapy

Published:2023-04 Issue:4 Volume:33 Page:309-314
ISSN:0960-8966
Container-title:Neuromuscular Disorders
language:en
Short-container-title:Neuromuscular Disorders

Author:

Barrois R.^ORCID,Barnerias C.,Deladrière E.,Leloup-Germa V.,Tervil B.,Audic F.,Boulay C.,Cances C.,Cintas P.,Davion J.B.,Espil-Taris C.,Manel V.,Pereon Y.,Piarroux J.,Quijano Roy S.,Vuillerot C.,Walther-Louvier U.,Desguerre I.,Gitiaux C.

Publisher

Elsevier BV

Subject

Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health

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4. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial;Day;Lancet Neurol,2021

5. European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy;Kirschner;Eur J Paediatr Neurol,2020

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4. Neurophysiological Characteristics in Type II and Type III 5q Spinal Muscular Atrophy Patients: Impact of Nusinersen Treatment;Drug Design, Development and Therapy;2024-03

5. Correlations between clinical motor scores and CMAP in patients with type 2 spinal muscular amyotrophy treated with nusinersen;Archives de Pédiatrie;2024-01