The Full Spectrum of Spinal Muscular Atrophy: From Diagnosis to Treatment
Med-IQ Select Med-IQ Select

The Full Spectrum of Spinal Muscular Atrophy: From Diagnosis to Treatment

Please choose a module from the options below. To receive credit and a certificate, you must complete all of the modules in this activity.

Welcome to this Med-IQ Select on spinal muscular atrophy (SMA). Med-IQ Select is a unique online educational offering that allows you to pick and choose the content you'd like to view—in any order and at your convenience.

To get started, click "view" on any of the modules below. To receive credit and a certificate, you must complete all of the modules.

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Overview of SMA

Time to Complete: 4 minutes

Genetics of SMA

Time to Complete: 5 minutes

Diagnosing SMA

Time to Complete: 6 minutes

Complete all of the above modules to earn credit;
0 out of 6 modules complete

Maximum Credits:

Released: 4/15/19

Expires: 4/14/20

Overview: This accredited CME publication provides an overview of the rare genetic disorder spinal muscular atrophy (SMA), including a review of SMA classification, genetic causes, clinical signs and differentiating factors from other motor neuron disorders, the role of genetic testing, and the importance of early diagnosis. It also explores the clinical rationale of genetic targets, new and emerging genetically focused disease-modifying treatments (ie, modulation of SMN2, SMN1 gene replacement, stabilization of the SMN protein), and patient- and family-centered care strategies. This publication also features audio clips that provide expert faculty insights on the various aspects of SMA and potential effects of new therapies on functional outcomes.

CME Information:

John Brandsema, MD
Assistant Professor of Clinical Neurology
Perelman School of Medicine
University of Pennsylvania
Attending Physician, Neuromuscular/General Neurology & Electromyography
Director, Neuromuscular Education
Neuromuscular Section Head
Children's Hospital of Philadelphia
Philadelphia, PA

Basil T. Darras, MD
Professor of Neurology
Harvard Medical School
Associate Neurologist-in-Chief
Chief, Division of Clinical Neurology
Director, Neuromuscular Center and Spinal Muscular Atrophy Program
Boston Children’s Hospital
Boston, MA

Activity Planners
Julie Blum, PhD
Director of Clinical Content
Baltimore, MD

Susan Kuhn, MHSc
Manager, Educational Strategy and Content
Baltimore, MD

Rebecca L. Julian, MS, ELS
Senior Manager, Editorial
Baltimore, MD

Samantha Gordon
CME Specialist
Baltimore, MD

Kathryn Schaefer, MSN, RN, CPHRM
Senior Manager, Accreditation and Compliance
East Lansing, MI

Katherine Kahn
Holyoke, MA

Learning Objective
Upon completion, participants should be able to:

  • Incorporate genetic testing into clinical practice for patients with suspected SMA
  • Use clinical evidence about recently approved or investigational therapies to guide treatment decisions, including consideration of clinical trial enrollment for appropriate patients with SMA

Target Audience
This activity is intended for neurologists, pediatricians, primary care physicians, genetic counselors, and neuromuscular specialists.
Statement of Need
SMA is a rare, autosomal recessive genetic disease characterized by muscle weakness and physical disability attributed to motor neuron degeneration in the spinal cord and brainstem. Early diagnosis is key because it may lead to early treatment initiation; however, delays in diagnosis are common in SMA. In light of the first therapy for SMA approved by the Food and Drug Administration, the prompt and accurate diagnosis is of great importance in the context of initiating early treatment. In addition, there is an active research pipeline filled with novel disease-modifying therapies that are producing promising results in clinical trials. These agents have the potential to progress to the mainstream relatively quickly; therefore, clinicians must have a thorough understanding of the efficacy and safety data and potential use of these agents so that they are prepared to incorporate them into clinical practice as they become available.
Providership Statement
Provided by Med-IQ.

Accreditation/Designation Statement
Med-IQ is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Med-IQ designates this enduring material for a maximum of 0.75 AMA PRA Category 1 Credit. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nurse practitioners, physician assistants, and other healthcare professionals who successfully complete the activity will receive a Statement of Participation indicating the maximum credits available.
Medium/Method of Participation
This CME activity consists of a 0.75-credit online publication. To receive credit, read the introductory CME material, complete all of the modules, and complete the evaluation, attestation, and post-test, answering at least 70% of the post-test questions correctly.
Initial Release Date: April 15, 2019
Expiration Date: April 14, 2020
Estimated Time to Complete This Activity: 45 minutes
Disclosure Policy
Med-IQ requires any person in a position to control the content of an educational activity to disclose all relevant financial relationships with any commercial interest. The ACCME defines “relevant financial relationships” as those in any amount occurring within the past 12 months, including those of a spouse/life partner, that could create a conflict of interest (COI). Individuals who refuse to disclose will not be permitted to contribute to this CME activity in any way. Med-IQ has policies in place that will identify and resolve COIs prior to this educational activity. Med-IQ also requires faculty to disclose discussions of investigational products or unlabeled/unapproved uses of drugs or devices regulated by the US Food and Drug Administration.
Disclosure Statement
The content of this activity has been peer reviewed and has been approved for compliance. The faculty and contributors have indicated the following financial relationships, which have been resolved through an established COI resolution process, and have stated that these reported relationships will not have any impact on their ability to give an unbiased presentation.
John Brandsema, MD
Consulting fees/advisory boards: Alexion, AveXis, Inc., Biogen Idec, Cytokinetics, Inc., PTC Therapeutics, Sarepta Therapeutics
Fees received for promotional/non-CME activities: Biogen Idec
Contracted research: Alexion, AveXis, Inc., Biogen Idec, Cytokinetics, Inc., PTC Therapeutics, Sarepta Therapeutics

Basil T. Darras, MD
Royalty: UpToDate, Elsevier
Consulting fees/advisory boards: AveXis, Inc., Biogen Idec, Cytokinetics, Inc., Dynacure, F. Hoffman-La Roche Ltd., PTC Therapeutics, Sarepta Therapeutics
Fees received for promotional/non-CME activities: Biogen Idec

Susan Kuhn, MHSc
Spouse: Ownership interest (stocks/stock options – excluding mutual funds): Illumina, Johnson & Johnson, Viking Therapeutics

The writer, peer reviewers, and other activity planners have no financial relationships to disclose.

Statement of Evidence-Based Content
Educational activities that assist physicians in carrying out their professional responsibilities more effectively and efficiently are consistent with the ACCME definition of continuing medical education (CME). As an ACCME-accredited provider of CME, it is the policy of Med-IQ to review and ensure that all the content and any recommendations, treatments, and manners of practicing medicine in CME activities are scientifically based, valid, and relevant to the practice of medicine. Med-IQ is responsible for validating the content of the CME activities it provides. Specifically, (1) all recommendations addressing the medical care of patients must be based on evidence that is scientifically sound and recognized as such within the profession; (2) all scientific research referred to, reported, or used in CME in support or justification of a patient care recommendation must conform to generally accepted standards of experimental design, data collection, and analysis.
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Acknowledgment of Commercial Support
This activity is supported by an educational grant from Genentech.

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