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Current Treatment Options in Neurology—SMA Therapeutics

  • Neuromuscular Disorders (C Fournier, Section Editor)
  • Published:
Current Treatment Options in Neurology Aims and scope Submit manuscript

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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Acknowledgments

We are grateful to Jill Jareki, PhD, and Jackie Glascock, PhD, from CureSMA for providing us with an updated list of the SMA Therapeutic Pipeline for use in this review.

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Authors and Affiliations

  1. Center for Gene Therapy, Nationwide Children’s Hospital, Columbus, OH, USA

    Megan A. Waldrop MD

  2. Department of Pediatrics, The Ohio State University Wexner Medical Center, Columbus, OH, USA

    Megan A. Waldrop MD

  3. Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA

    Megan A. Waldrop MD & Stephen J. Kolb MD, PhD

  4. Department of Biological Chemistry & Pharmacology, The Ohio State University Wexner Medical Center, Columbus, OH, USA

    Stephen J. Kolb MD, PhD

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  1. Megan A. Waldrop MD
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Correspondence to Stephen J. Kolb MD, PhD.

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Conflict of Interest

Stephen J. Kolb reports consulting fees from AveXis, Biogen Idec, and Genentech outside the submitted work. Megan A. Waldrop reports personal fees from The France Foundation outside the submitted work.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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Waldrop, M.A., Kolb, S.J. Current Treatment Options in Neurology—SMA Therapeutics. Curr Treat Options Neurol 21, 25 (2019). https://doi.org/10.1007/s11940-019-0568-z

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