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

[en] 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.

Disciplines :

Neurology

Author, co-author :

Dangouloff, Tamara ; Université de Liège - ULiège > Département des sciences cliniques > Neuropédiatrie

Servais, Laurent ; Université de Liège - ULiège > Département des sciences cliniques > Département des sciences cliniques

Language :

English

Title :

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

Publication date :

2019

Journal title :

Therapeutics and Clinical Risk Management

ISSN :

1176-6336

eISSN :

1178-203X

Publisher :

Dove Medical Press, Auckland, New Zealand

Volume :

Pages :

1153-1161

Peer reviewed :

Peer Reviewed verified by ORBi

Commentary :

Available on ORBi :

since 22 October 2019

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53 (7 by ULiège)

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2 (2 by ULiège)

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Scopus citations^®

130

Scopus citations^®
without self-citations

OpenCitations

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