Future Perspectives in SMA Management – Current and Emerging Strategies

This webcast was recorded during a satellite symposium at the 46th Child Neurology Society (CNS) Annual Meeting in Kansas, MO, USA, on October 6, 2017. During the symposium, the faculty of leading KOLs in the field provided an overview of the genetics and biology of spinal muscular atrophy (SMA) and addressed the subject of the importance of early diagnosis and accurate classification. The use of gene profiling and a review of the current best practices in multidisciplinary management were included in the program, and were illustrated with selected patient case presentations. The panel of experts discussed unmet needs in SMA management, and examined emerging treatment options based on the latest scientific data; they used plenary presentations to encourage the exchange of views and opinions on the rapidly evolving SMA treatment landscape.


This activity has been designed to meet the educational needs of health-care professionals involved in the diagnosis, treatment, or management of patients with SMA.


Upon completion of this activity, participants will be better able to do the following:

  1. Recommend SMA gene profiling prior to other testing as the most efficient means of diagnosing SMA
  2. Classify SMA type when evaluating patients, based on neurological examination and gene profiling
  3. Select and appropriately use the standardized assessment tools/tests to monitor for clinical changes in patients with SMA
  4. List key unmet needs in the management of SMA and understand how these gaps are being addressed with novel treatments
  5. Describe the benefits of the first treatment approved for the management of SMA
  6. Identify possible roles of other treatments currently in development, and identify patients who would most probably benefit from these therapies
  7. Develop a comprehensive care plan with parents and patients, in close cooperation with neurologists, physical therapists, respiratory therapists, and other members of the multidisciplinary team


John Day, MD, PhD
Department of Neurology
Stanford University School of Medicine
Stanford, CA, USA

Thomas Crawford, MD
MDA Clinic for Neuromuscular Disorders and Ataxia Telangiectasia Clinical Center
Johns Hopkins Hospital
Baltimore, MD, USA

Russell Butterfield, MD, PhD
Department of Pediatrics and Department of Neurology
University of Utah
Salt Lake City, UT, USA

Disclosure of Conflicts of Interest

It is the policy of the Elsevier Office of Continuing Medical Education that all faculty, instructors, and planners disclose any real or apparent conflicts of interest relating to the topics of this educational activity.

The faculty reported the following financial relationships or relationships to products or devices they or their life partner have with commercial interests related to the content of this interprofessional educational activity:

FacultyRelationship Identified With:

John Day, MD, PhD

Consultant/Advisor: Biogen, AveXis, Inc., Cytokinetics Inc.

Grant/Research Support: Biogen, AveXis, Inc., Cytokinetics Inc., F. Hoffman-La Roche Ltd

Thomas Crawford, MD

Consultant/Advisor: Biogen, AveXis, Inc.

Russell Butterfield, MD, PhD

Consultant/Advisor: Biogen, WAVE Life Sciences

Non-faculty: Sandy Breslow; Alison Kemp; Bernard M. Abrams, MD; Cordelia Nagle; Elena Knoche; Mirella Mels; Nabil Dorkhom; Marijke Verhaaf; hereby state that neither they nor their spouse/life partner have had any financial relationships to products or devices with any commercials interest related to the content of this activity of any amount during the past 12 months.

Financial Support

This activity has been supported by an independent educational grant from Biogen.

Provider Information

Jointly provided by the Elsevier Office of Continuing Medical Education and Excerpta Medica.

CME Credit (Physicians)

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of the Elsevier Office of Continuing Medical Education and Excerpta Medica. The Elsevier Office of Continuing Medical Education is accredited by the ACCME to provide continuing medical education for physicians.

The Elsevier Office of Continuing Medical Education designates this enduring activity for a maximum of 1.25 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.


For all CME inquiries or special needs, please contact elsevierCME@elsevier.com.

Disclosure of Unlabeled Use: This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The Elsevier Office of Continuing Medical Education, Excerpta Medica and Biogen do not recommend the use of any agent outside of the labeled indications.

Disclaimer: Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications on dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

Course summary
Available credit: 
  • 1.25 AMA PRA Category 1 Credit(s)™
  • 1.25 Non-physician
Course opens: 
Course expires: 

Available Credit

  • 1.25 AMA PRA Category 1 Credit(s)™
  • 1.25 Non-physician
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