Disease Modifying Therapies for the Management of Children with Spinal Muscular Atrophy (5q SMA): An Update on the Emerging Evidence

Disease Modifying Therapies for the Management of Children with Spinal Muscular Atrophy (5q SMA): An Update on the Emerging Evidence

Helgi Thor Hjartarson,1 Kristofer Nathorst-Böös,1 Thomas Sejersen1,2 1Department of Neuropediatrics, Astrid Lindgren Children´s Hospital, Karolinska University Hospital, Stockholm, Sweden; 2Department of Women’s and Children’s Health, Karolinska Institute, Stockholm, SwedenCorrespondence: Thomas Sej...

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Bibliographic Details
Main Authors: Hjartarson HT, Nathorst-Böös K, Sejersen T
Format: Article
Language:English
Published: Dove Medical Press 2022-06-01
Series:Drug Design, Development and Therapy
Subjects:
spinal muscular atrophy
treatment
disease-modifying
gene therapy
Online Access:https://www.dovepress.com/disease-modifying-therapies-for-the-management-of-children-with-spinal-peer-reviewed-fulltext-article-DDDT

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