The content for this activity is based on the satellite symposium, “Joining a Conversation on Improving the Multidisciplinary Management of SMA: Now and in the Future”, that was presented at the 45th Child Neurology Society Annual Meeting in Vancouver, Canada, on October 27, 2016.
Spinal muscular atrophy (SMA) is a complex neurodegenerative disease with a profile that requires a multidisciplinary approach to diagnosis and treatment, caused by mutations in the survival motor neuron 1 (SMN1) gene. There is currently no cure for SMA. Treatment strategy is based on the current understanding of the history of the condition combined with patient genetic profiles, as well as an integrated approach to recognizing the signs and symptoms frequently observed in patients with SMA. The key to continued progress in the treatment of SMA is found in the acceptance of new and emerging therapeutic strategies that will enable physicians to take a collaborative approach to patient treatment, managing the condition through its natural course.
This presentation will provide an overview of the clinical picture of SMA and its natural history, offer guidance on the diagnosis and classification of SMA by neurological examination and gene profiling, discuss the appropriate use of available assessment tools and outcomes measures, and review the emerging treatment landscape via the latest scientific data. Using interactive plenary sessions and selected patient case presentations, our expert panel will 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 spinal muscular atrophy.
Upon completion of this activity, participants will be better able to do the following:
- Describe the typical clinical picture of SMA and its natural history, and differentiate between SMA types on the basis of neurological examination and gene profiling
- Select and appropriately use standardized assessment tools and tests to monitor for clinical changes and disease progression
- Understand the modes of action and clinical profiles of emerging therapies and their possible roles in the modern treatment paradigm
- Develop a comprehensive, multidisciplinary care plan and know how to modify the therapeutic approach as SMA progresses
Richard S. Finkel, MD (Chair)
University of Central Florida School of Medicine
Nemours Children’s Hospital
Orlando, FL, USA
Basil T. Darras, MD
Harvard Medical School
Boston Children’s Hospital
Boston, MA, USA
Amy Pasternak, DPT
Boston Children’s Hospital
Boston, MA, USA
Perry Shieh, MD, PhD
David Geffen School of Medicine at UCLA
Ronald Reagan UCLA Medical Center
Los Angeles, CA, 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 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 spouse/life partner have with commercial interests related to the content of this CME activity:
|Faculty||Relationship Identified With:|
Basil T. Darras, MD
Consultant/Advisor: AveXis, Inc.; Biogen; Bristol-Myers Squibb Company; Cytokinetics, Inc.; Marathon Pharmaceuticals, LLC; PTC Therapeutics
Grant/Research Support: Cytokinetics,Inc.; Ionis Pharmaceuticals; PTC Therapeutics; Sarepta Therapeutics
Richard S. Finkel, MD
Consultant/Advisor: AveXis, Inc.; Biogen; Hoffmann-La Roche Ltd; Ionis Pharmaceuticals; Novartis AG
Grant/Research Support: Biogen; Cytokinetics, Inc.; Hoffmann-La Roche Ltd; Ionis Pharmaceuticals
|Amy Pasternak, DPT||Consultant/Advisor: Biogen|
|Perry Shieh, MD, PhD|
Consultant/Advisor: AveXis, Inc.; Biogen; Genentech, Inc.; Novartis AG (non-SMA product)
Non-faculty: Lara Quigley; Marijke Verhaaf; Caroline van Geelen; Sandy Breslow; Alison Kemp; Leah Johnson; and Bernard M. Abrams, MD hereby state that neither they nor their spouse/life partner have had any financial relationships to products or devices with any commercial interest related to the content of this activity of any amount during the past 12 months.
This activity has been supported by an independent educational grant from Biogen.
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 1.5 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
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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.