Castro D, Finkel RS, Farrar MA, et al. Nusinersen in infantile-onset spinal muscular atrophy: results from longer-term treatment from the open-label SHINE extension study. Neurology. 2020;94(15 suppl): Abstract 1640.
Chand D, Mohr F, McMillan H, et al. Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy. J Hepatol. 2020. doi: 10.1016/j.jhep.2020.11.001. [Epub ahead of print].
Chiriboga CA, Darras BT, Farrar MA, et al. Longer-term treatment with nusinersen: results in later-onset spinal muscular atrophy from the SHINE study (1661). Neurology. 2020;94(15 suppl): Abstract 1661.
cureSMA. Testing & diagnosis. Accessed January 14, 2021. https://www.curesma.org/testing-diagnosis/
Day JW, Swoboda KJ, Darras BT, et al. Longer-term experience with nusinersen in young adults with spinal muscular atrophy: results from the CS2/CS12 and SHINE studies. Neurology. 2020;94(15 suppl): Abstract 1132.
Day JW, Chiriboga CA, Crawford TO, et al. Onasemnogene abeparvovec-xioi gene-replacement therapy for spinal muscular atrophy type 1 (SMA1): phase 3 US study STR1VE update. Neurology. 2020;94(15 suppl): Abstract 1828
Kariyawasam DST, Russell JS, Wiley V, et al. The implementation of newborn screening for spinal muscular atrophy: the Australian experience. Genet Med. 2020;22:557-565.
Kay DM, Stevens CF, Parker A, et al. Implementation of population-based newborn screening reveals low incidence of spinal muscular atrophy. Genet Med. 2020;22:1296-1302.
Mercuri E, Baranello G, Masson R, et al. Onasemnogene aveparvovec [sic] gene therapy for spinal muscular atrophy type 1 (SMA1): phase 3 study update (STR1VE-EU). Neuromuscul Disord. 2020;30:S46-S150: Abstract P.258.
National Organization for Rare Diseases. Rare Disease Database. Spinal muscular atrophy. Accessed January 14, 2021. https://rarediseases.org/rare-diseases/spinal-muscular-atrophy/
Pera MC, Coratti G, Berti B, et al. Diagnostic journey in spinal muscular atrophy: is it still an odyssey? PLoS One. 2020;15:e0230677.
Ramdas S, Servais L. New treatments in spinal muscular atrophy: an overview of currently available data. Expert Opin Pharmacother. 2020;21:307-315.
Swoboda KJ, Kirschner J, Finkel RS, on behalf of the NURTURE Study Group. Nusinersen effect in infants who initiate treatment in a presymptomatic stage of SMA: NURTURE results. Presented at: Cure Spinal Muscular Atrophy (CSMA) 2020, June 11-14, 2020; Orlando, FL.
US Food & Drug Administration. FDA approves oral treatment for spinal muscular atrophy. Published August 7, 2020. Accessed January 14, 2021. https://www.fda.gov/news-events/press-announcements/fda-approves-oral-treatment-spinal-muscular-atrophy
US Food & Drug Administration. FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. Released May 24, 2019. Accessed January 14, 2021. https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease

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Faculty

Richard Shell, MD

Department of Pulmonary Medicine
Nationwide Children's Hospital and
Ohio State University College of Medicine
Columbus, Ohio

Disclosures

Disclosure: Richard Shell, MD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Avexis/Novartis

Nancy Kuntz, MD

Mazza Foundation Neuromuscular Program
Ann & Robert H. Lurie Children's Hospital of Chicago
Muscular Dystrophy Association Care Center
Northwestern University Feinberg School Medicine
Chicago, Illinois

Disclosures

Disclosure: Nancy Kuntz, MD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Argenyx; Audentes; Biogen; Genentech; Novartis; Sarepta
Received grants for clinical research from: Audentes; Biogen; Novartis; Sarepta

Editors

Christina T. Loguidice, BA

Medical Education Director, Medscape, LLC

Disclosures

Disclosure: Christina T. Loguidice, BA, has disclosed no relevant financial relationships.

Rosalyn Blumenthal, PhD

Scientific Content Manager, Medscape, LLC

Disclosures

Disclosure: Rosalyn Blumenthal, PhD, has disclosed no relevant financial relationships.

Heather Lewin, MAT

Director, Medscape, LLC

Disclosures

Disclosure: Heather Lewin, MAT, has disclosed no relevant financial relationships.

CME Reviewer

Robert Morris, PharmD

Associate Director, Accreditation and Compliance, Medscape, LLC

Disclosures

Disclosure: Robert Morris, PharmD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: ViiV Healthcare
Owns stock, stock options, or bonds from: GlaxoSmithKline

Medscape, LLC staff have disclosed that they have no relevant financial relationships.

Peer Reviewer

This activity has been peer reviewed and the reviewer has disclosed no relevant financial relationships.

CME

2020 Updates in Spinal Muscular Atrophy: A Review of What's New

Authors: Richard Shell, MD; Nancy Kuntz, MD
CME Released: 1/26/2021
THIS ACTIVITY HAS EXPIRED FOR CREDIT
Valid for credit through: 1/26/2022, 11:59 PM EST

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Target Audience and Goal Statement

This activity is intended for neurologists, primary care physicians, and pediatricians.

The goal of this activity is to inform clinicians regarding 2020 clinical data related to the diagnosis and management of spinal muscular atrophy (SMA).

Upon completion of this activity, participants will:

Have increased knowledge regarding the
- New data related to the diagnosis of SMA
- New data related to the management of SMA

Disclosures

Faculty

Richard Shell, MD

Department of Pulmonary Medicine
Nationwide Children's Hospital and
Ohio State University College of Medicine
Columbus, Ohio

Disclosures

Disclosure: Richard Shell, MD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Avexis/Novartis

Nancy Kuntz, MD

Mazza Foundation Neuromuscular Program
Ann & Robert H. Lurie Children's Hospital of Chicago
Muscular Dystrophy Association Care Center
Northwestern University Feinberg School Medicine
Chicago, Illinois

Disclosures

Disclosure: Nancy Kuntz, MD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Argenyx; Audentes; Biogen; Genentech; Novartis; Sarepta
Received grants for clinical research from: Audentes; Biogen; Novartis; Sarepta

Editors

Christina T. Loguidice, BA

Medical Education Director, Medscape, LLC

Disclosures

Disclosure: Christina T. Loguidice, BA, has disclosed no relevant financial relationships.

Rosalyn Blumenthal, PhD

Scientific Content Manager, Medscape, LLC

Disclosures

Disclosure: Rosalyn Blumenthal, PhD, has disclosed no relevant financial relationships.

Heather Lewin, MAT

Director, Medscape, LLC

Disclosures

Disclosure: Heather Lewin, MAT, has disclosed no relevant financial relationships.

CME Reviewer

Robert Morris, PharmD

Associate Director, Accreditation and Compliance, Medscape, LLC

Disclosures

Disclosure: Robert Morris, PharmD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: ViiV Healthcare
Owns stock, stock options, or bonds from: GlaxoSmithKline

Medscape, LLC staff have disclosed that they have no relevant financial relationships.

Peer Reviewer

This activity has been peer reviewed and the reviewer has disclosed no relevant financial relationships.

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From Medscape Education Neurology & Neurosurgery

CME

2020 Updates in Spinal Muscular Atrophy: A Review of What's New

Authors: Richard Shell, MD; Nancy Kuntz, MDFaculty and Disclosures

THIS ACTIVITY HAS EXPIRED FOR CREDIT

CME Released: 1/26/2021

Valid for credit through: 1/26/2022, 11:59 PM EST

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Disclaimer

The educational activity presented above may involve simulated, case-based scenarios. The patients depicted in these scenarios are fictitious and no association with any actual patient, whether living or deceased, is intended or should be inferred. The material presented here does not necessarily reflect the views of Medscape, LLC, or any individuals or commercial entities that support companies that support educational programming on medscape.org. These materials may include discussion of therapeutic products that have not been approved by the US Food and Drug Administration, off-label uses of approved products, or data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal. Readers should verify all information and data before treating patients or employing any therapies described in this or any educational activity. A qualified healthcare professional should be consulted before using any therapeutic product discussed herein.

References

Faculty and Disclosures

Faculty

Richard Shell, MD

Nancy Kuntz, MD

Editors

Christina T. Loguidice, BA

Rosalyn Blumenthal, PhD

Heather Lewin, MAT

CME Reviewer

Robert Morris, PharmD

Peer Reviewer

CME

2020 Updates in Spinal Muscular Atrophy: A Review of What's New

Target Audience and Goal Statement

Disclosures

Faculty

Richard Shell, MD

Nancy Kuntz, MD

Editors

Christina T. Loguidice, BA

Rosalyn Blumenthal, PhD

Heather Lewin, MAT

CME Reviewer

Robert Morris, PharmD

Peer Reviewer

Accreditation Statements

For Physicians

Instructions for Participation and Credit

2020 Updates in Spinal Muscular Atrophy: A Review of What's New