Article (Scientific journals)
New treatments in spinal muscular atrophy: an overview of currently available data
Ramdas, S.; Servais, Laurent
2020In Expert Opinion on Pharmacotherapy, 21 (3), p. 307-315
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Keywords :
Spinal muscular atrophy
Abstract :
[en] Introduction: Spinal muscular atrophy (SMA) is one of the most common inherited neuromuscular disorders. It causes progressive muscle weakness and results in significant disability. Until recently, there were no drugs available for the treatment of SMA. Several phase 1-3 studies, including three double-blind randomized placebo-controlled studies have demonstrated the efficacy of disease-modifying approaches including gene replacement therapy, antisense oligonucleotides, and splicing modifiers.Areas covered: This article covers the publically available data on therapeutic strategies that address the underlying cause of SMA and clinical data available on approved treatments and drugs in the pipeline.Expert opinion: The newer therapeutic options in SMA have a good safety profile and deliver a therapeutic benefit in most patients. It is essential that the recommended standards of care are delivered along with the drugs for the best outcomes. No biomarkers to distinguish responders from non-responders are available; it is important that biomarkers be identified. Early treatment is essential for the maximum efficacy of the newly available treatments.
Disciplines :
Neurology
Pediatrics
Author, co-author :
Ramdas, S.;  MDUK Neuromuscular Center, Department of Paediatrics, University of Oxford, Oxford, United Kingdom
Servais, Laurent ;  Université de Liège - ULiège > Département des sciences cliniques > Département des sciences cliniques
Language :
English
Title :
New treatments in spinal muscular atrophy: an overview of currently available data
Publication date :
February 2020
Journal title :
Expert Opinion on Pharmacotherapy
ISSN :
1465-6566
eISSN :
1744-7666
Publisher :
NLM (Medline)
Volume :
21
Issue :
3
Pages :
307-315
Peer reviewed :
Peer Reviewed verified by ORBi
Available on ORBi :
since 01 February 2021

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