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Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives

Authors Dangouloff T, Servais L

Received 27 July 2019

Accepted for publication 8 September 2019

Published 2 October 2019 Volume 2019:15 Pages 1153—1161

DOI https://doi.org/10.2147/TCRM.S172291

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Nicola Ludin

Peer reviewer comments 2

Editor who approved publication: Professor Garry Walsh


Tamara Dangouloff,1 Laurent Servais1,2

1Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège & University of Liège, Liège, Belgium; 2MDUK Neuromuscular Center, Department of Paediatrics, University of Oxford, Oxford, UK

Correspondence: Laurent Servais
CRMN, CHU Liège, Boulevard du Douzième de Ligne, 1, Liège 4000, Belgium
Tel +33 6 22 59 22 43
Fax +32 4 321 83 28
Email lservais@chuliege.be

Abstract: Recent advances in the treatment of spinal muscular atrophy (SMA) have dramatically altered prognosis. Rather than a rapidly lethal disease, SMA type 1, the most severe form with the earliest onset of SMA, has become a disease in which long-term event-free survival with the acquisition of important motor milestones is likely. Prognosis for patients with SMA type 2 has shifted from slow and progressive deterioration to long-term stability. Nevertheless, there is a large heterogeneity in terms of clinical response to currently available treatments, ranging from absence of response to impressive improvement. The only factor identified that is predictive of treatment success is the age of the patient at the initiation of treatment, which is closely related to disease duration. The aim of this paper is to review available evidence that support early intervention using currently available treatment approaches.

Keywords: spinal muscular atrophy, nusinersen, zolgensma, risdiplam, branaplam, newborn screening

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