Therapy for Spinal Muscular Atrophy

To the Editor: In two reports regarding patients with type 1 spinal muscular atrophy (Nov. 2 issue), 1,2 researchers describe therapies involving antisense oligonucleotides (Finkel et al.) and gene therapy (Mendell et al.). The editorial by van der Ploeg 3 draws attention to differences in the desig...

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Bibliographic Details
Published inThe New England journal of medicine Vol. 378; no. 5; pp. 487 - 488
Main Authors Whittaker, Heather T, Michell-Robinson, Mackenzie A, Mendell, Jerry R, Finkel, Richard S, Farwell, Wildon
Format Journal Article
LanguageEnglish
Published United States Massachusetts Medical Society 01.02.2018
Subjects
Humans
Muscular Atrophy, Spinal
Spinal Muscular Atrophies of Childhood
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Summary:To the Editor: In two reports regarding patients with type 1 spinal muscular atrophy (Nov. 2 issue), 1,2 researchers describe therapies involving antisense oligonucleotides (Finkel et al.) and gene therapy (Mendell et al.). The editorial by van der Ploeg 3 draws attention to differences in the designs of the two studies. In particular, the differences in the ages of infants who were enrolled in each study make it difficult to compare the efficacy of treatments. We wonder whether Finkel et al. have data regarding infants who were treated at ages that were similar to those of infants treated by Mendell et al. . . .
Bibliography:SourceType-Other Sources-1
content type line 63
ObjectType-Correspondence-1
ObjectType-Commentary-2
ISSN:0028-4793
1533-4406
DOI:10.1056/NEJMc1715769