Advancing Team-Based Care for Spinal Muscular Atrophy

Advancing Team-Based Care for Spinal Muscular Atrophy

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Developed in collaboration
Med-IQ      StanfordMedicineCME

Online Course | Specialties: Neurology, Pediatrics
Released: 4/27/2022
Expires: 2/26/2023
Max Credits: 1.0
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Jenna Klotz, MD, MS
Clinical Assistant Professor, Neurology & Neurological Sciences and Pediatrics - Operations
Stanford University School of Medicine
Pediatric Neurologist
Stanford Health Care-Stanford Hospital
Lucile Packard Children’s Hospital
Palo Alto, CA

Ana Carolina Tesi-Rocha, MD
Clinical Associate Professor, Neurology & Neurological Sciences and Pediatrics - Operations
Stanford University School of Medicine
Pediatric Neurologist
Lucile Packard Children’s Hospital
Palo Alto, CA

Activity Planners
Lisa Hack
Manager, Education Strategy and Content
Baltimore, MD

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

Samantha Gordon, MS
Accreditation Manager
Baltimore, MD

Amy Sison
Director of CME
Baltimore, MD

Kathryn Schaefer, MSN, RN, CPHRM, CHCP, FASHRM
Associate Director, Education Quality and Compliance
East Lansing, MI

Ruth Adewuya, MD, CHCP
Managing Director
Center for Continuing Medical Education
Stanford University School of Medicine
Palo Alto, CA

Katherine Kahn
Holyoke, MA

Learning Objectives
Upon completion, participants should be able to:
  • Summarize effective strategies for the provision of well-coordinated, multidisciplinary care for patients with SMA
  • Integrate genetic testing into clinical practice for patients with suspected SMA
  • Incorporate the latest findings with novel therapies into treatment decisions for patients with SMA, including in the context of clinical trial enrollment for appropriate patients

Target Audience
This activity is intended for multidisciplinary teams of neurologists, genetic counselors, nurses, neuromuscular specialists, social workers, and specialty care providers who manage patients with SMA.

Statement of Need
Spinal muscular atrophy (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. The early diagnosis of SMA is key because it may lead to early treatment initiation, but delays in diagnosis commonly occur. Updated guidelines for care reiterate the critical need for multidisciplinary, patient-centered care to improve patient outcomes and quality of life; however, effective team-based care coordination can sometimes prove to be challenging. Novel treatment options are now available for SMA, and there is an active research pipeline of emerging therapies, with clinical trials providing data on the efficacy, safety, and potential use of these agents. Healthcare providers must be prepared to incorporate these treatment options safely and appropriately into clinical practice as they become available.

Collaboration Statement
This activity was developed by Med-IQ in collaboration with the Stanford Center for Continuing Medical Education.

Accreditation/Designation Statements
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 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Med-IQ is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center’s Commission on Accreditation.

This nursing activity has been approved for up to 1.0 contact hour.

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/CE activity consists of a 1.0-credit online publication. To receive credit, read the introductory CME/CE material, read the publication, and complete the post-survey, evaluation, attestation, and post-test, answering at least 70% of the post-test questions correctly.

Initial Release Date: April 27, 2022
Expiration Date: April 26, 2023
Estimated Time to Complete This Activity: 1 hour

Disclosure Policy
Med-IQ requires any person in a position to control the content of an educational activity to disclose all financial relationships with any ineligible company over the past 24 months. The ACCME deems financial relationships as relevant if the educational content an individual can control is related to the business lines or products of the ineligible company. 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 mitigate 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.

Drug/Product Usage by Faculty
Off-label/unapproved drug uses or products are mentioned within this activity.

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 mitigated through an established COI mitigation process, and have stated that these reported relationships will not have any impact on their ability to give an unbiased presentation.

Jenna Klotz, MD, MS
Consulting fees/advisory boards: F. Hoffmann-La Roche Ltd., Genentech, Sarepta Therapeutics

Ana Carolina Tesi-Rocha, MD
Consulting fees/advisory boards: AveXis, Biogen Idec, Genentech, Novartis Pharmaceuticals Corporation

The writer, peer reviewers, and 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, Med-IQ has a policy 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.

Med-IQ is not liable for any decision made or action taken in reliance upon the information provided through this activity.

Contact Information
For other questions or comments about this activity, please contact Med-IQ. Call (toll-free) 866 858 7434 or email


System Requirements
For technical assistance, please refer to our Support Manual.

The information provided through this activity is for continuing education purposes only and is not meant to substitute for the independent medical judgment of a physician relative to diagnostic and treatment options of a specific patient’s medical condition.

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Complimentary CME/CE
This activity is available free of charge to participants.

Acknowledgment of Commercial Support
This activity is supported by an educational grant from Genentech.


Here are the key takeaways from this activity. Deeper insights and evidence, plus an opportunity to receive credit, are available at the "Continue" button below.

  • SMA is caused by a mutation in the survival of motor neuron gene 1 (SMN1) that impairs the body’s ability to produce a sufficient amount of SMN protein, which in turn leads to debilitating and sometimes fatal muscle weakness
  • Optimal management of SMA includes neuromuscular and musculoskeletal evaluation and rehabilitation; orthopedic management of any spinal deformities, joint dislocations, and contractures; gastrointestinal and nutritional management; and pulmonary management; as well as consideration of anticipatory care plans, ethical issues, and palliative care
  • Three medications are currently available for the treatment of SMA—nusinersen (an antisense oligonucleotide), risdiplam (a small molecule splice modifier), and onasemnogene abeparvovec-xioi (a form of gene-replacement therapy); with no evidence to support the superiority of one treatment over another, the primary factors guiding treatment choice include age of onset, current functional status, the presence of scoliosis or contractures, AAV9 antibody status, and the number of SMN2 copies
  • Regular monitoring of patients is important to document changes over time, provide a comparison for trajectories of progression reported in clinical trials and other research, identify needs for intervention, and assess responses to interventions
  • SMA is a chronic illness with profound ramifications for the entire family; case managers and social workers are often instrumental in educating and supporting the family and helping with coordinating care, managing cost/insurance issues, developing individual education plans, and navigating life transitions, as well as helping caregivers meet their own needs

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Unless otherwise indicated, photographed subjects who appear within the content of this activity or on artwork associated with this activity are models; they are not actual patients or doctors.

The information provided through this activity is for continuing education purposes only and is not meant to substitute for the independent medical judgment of a physician relative to diagnostic and treatment options of a specific patient’s medical condition.

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