Diagnosis and Treatment of Spinal Muscular Atrophy in an Evolving Therapeutic Landscape
Presentation Points Presentation Points

Diagnosis and Treatment of Spinal Muscular Atrophy in an Evolving Therapeutic Landscape


Released:
1/23/20
Expires:
1/22/21

Maximum Credits:

1.5
By clicking "Continue," you are confirming that you have reviewed the CME information and read, understood, and unconditionally agreed to the Privacy Notice and Terms of Use.

Presentation Points Presentation Points
By clicking "Continue," you are confirming that you have reviewed the CME information and read, understood, and unconditionally agreed to the Privacy Notice and Terms of Use.
Released:
1/23/20

Expires:
1/22/21

Maximum Credits:
1.5
Presentation Points Presentation Points
By clicking "Continue," you are confirming that you have reviewed the CME information and read, understood, and unconditionally agreed to the Privacy Notice and Terms of Use.

Released:
1/23/20
Expires:
1/22/21


Maximum Credits:
1.5


Overview: This transcript-based, CME-accredited 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 causes of muscle weakness, the role of genetic testing, and the importance of early diagnosis. It also explores patient- and family-centered care strategies and the potential effects of new therapies on functional outcomes.

CME Information:

Faculty
John Brandsema, MD
Assistant Professor of Clinical Neurology
Perelman School of Medicine
University of Pennsylvania
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

Richard S. Finkel, MD
Professor of Neurology 
University of Central Florida College of Medicine
Chief, Division of Pediatric Neurology 
Director, Neuromuscular Program 
Nemours Children’s Hospital 
Orlando, FL 

Activity Planners
Julie Blum, PhD
Director, Clinical Content
Med-IQ
Baltimore, MD

Laura Rafferty, ELS
Managing Editor
Med-IQ
Baltimore, MD

Samantha Gordon
CME Specialist
Med-IQ
Baltimore, MD

Kathryn Schaefer, MSN, RN, CPHRM
Senior Manager, Accreditation and Compliance
Med-IQ
East Lansing, MI 
 
Learning Objectives
Upon completion, participants should be able to: 

  • Describe symptoms of SMA in an infant or child 
  • Discuss the benefits of early diagnosis and treatment of SMA
  • Understand the role of pediatricians in maintaining best practices for treating SMA
  • Describe current newborn screening standards for SMA 

Target Audience
This activity is intended for physicians (including pediatricians, pediatric neurologists, and neuromuscular specialists), nurse practitioners, physician assistants, nurses, and other clinicians who care for people with SMA.
 
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 recent United States Food and Drug Administration approvals of therapies for SMA, clinicians must be able to promptly and accurately diagnose this condition. Moreover, there is an active research pipeline that includes several novel disease-modifying therapies that are producing promising results in clinical trials. These agents have the potential to transition to mainstream clinical practice relatively quickly; therefore, clinicians must have a thorough understanding of the efficacy and safety data and potential role of these agents so that they are prepared to incorporate them into treatment regimens as they become available. 

Providership Statement
Provided by Med-IQ.

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 1.5 AMA PRA Category 1 Credits™. 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 1.5-credit online publication. To receive credit, read the introductory CME 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: January 23, 2020
Expiration Date: January 22, 2021
Estimated Time to Complete This Activity: 1 hour and 30 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: AveXis, Biogen Idec, Cytokinectics, Inc., PTC Therapeutics 
Fees received for promotional/non-CME activities: Biogen Idec
Contracted research: Alexion Pharmaceuticals, Inc., AveXis, Biogen Idec, Cytokinectics, Inc., FibroGen, Inc., Pfizer, Inc., PTC Therapeutics, Sarepta Therapeutics, Wave 

Basil T. Darras, MD
Consulting fees/advisory boards: AveXis, Biogen Idec, Cytokinetics, Inc., Dynacure, F. Hoffmann-La Roche Ltd., Genentech, Sarepta Therapeutics  

Richard S. Finkel, MD
Consulting fees/advisory boards: AveXis, Biogen Idec, Cytokinetics, Inc., F. Hoffmann-La Roche Ltd., Genentech, Neurogene Inc., Novartis Pharmaceuticals Corporation, Scholar Rock
Contracted research: AveXis, Biogen Idec, Catabasis Pharmaceuticals, Inc., Cytokinetics, Inc., F. Hoffmann-La Roche Ltd., Italfarmaco S.p.A., ReveraGen BioPharma, Inc., Scholar Rock, Summit Pharmaceuticals International Corporation 

The 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 questions or comments about this activity, please contact Med-IQ. Call (toll-free) 866 858 7434 or email info@med-iq.com.          
                      
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Mobile

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

Copyright
© 2020 Med-IQ, Inc.

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.

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