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Current Treatment Options in Neurology

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01-06-2019 | Spinal Muscular Atrophy | Neuromuscular Disorders (C Fournier, Section Editor)

Current Treatment Options in Neurology—SMA Therapeutics

Authors: Megan A. Waldrop, MD, Stephen J. Kolb, MD, PhD

Published in: Current Treatment Options in Neurology | Issue 6/2019

Abstract

Purpose of review

In this review, we discuss the clinical and genetic features of 5q spinal muscular atrophy and highlight approved and upcoming therapies.

Recent findings

We emphasize that multidisciplinary care has been a key component of the improved quality and length of life seen in these individuals in the past decade. We discuss the evidence leading to the approval of nusinersen and the evidence leading to the anticipated approval of onasemnogene abeparvovec-xioi. Additional clinical therapies that are on the horizon are discussed and the importance of continued multidisciplinary care even after treatment is emphasized.

Summary

The pursuit of therapies for spinal muscular atrophy is becoming a success story and continued development of biomarkers will allow for more informed therapeutic decision making and eventual cost-effective utilization of available therapies.

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Title: Current Treatment Options in Neurology—SMA Therapeutics
Authors: Megan A. Waldrop, MD
Stephen J. Kolb, MD, PhD
Publication date: 01-06-2019
Publisher: Springer US
Keywords: Spinal Muscular Atrophy
Gene Therapy in Oncology
Published in: Current Treatment Options in Neurology / Issue 6/2019
Print ISSN: 1092-8480
Electronic ISSN: 1534-3138
DOI: https://doi.org/10.1007/s11940-019-0568-z

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Springer Medicine

Current Treatment Options in Neurology—SMA Therapeutics

Abstract

Purpose of review

Recent findings

Summary

At a glance: The ONWARDS insulin icodec trials

Springer Medicine

Abstract

Purpose of review

Recent findings

Summary

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