An Interactive Workshop to Improve the Journey to Adulthood in Spinal Muscular Atrophy

Philadelphia, PA US
October 26, 2023

Program Description

Historically, patients with the most severe forms of SMA were largely unable to survive into adulthood and those that did often did not continue to see neurologists. However, with the arrival of therapies that can dramatically shift the path of the disease, more and more patients are entering adult care. Unfortunately, as this transition is a new consideration, many clinicians are still learning how to coordinate and manage it appropriately. As such, this program is designed to bring together clinicians from both sides of these transitions to glean clinical pearls from leaders in the space and the established programs at CHOP and the University of Pennsylvania while opening the floor to attendees to voice their own challenges and solutions in this process. 

Target Audience

This program is intended for general neurologists, neuromuscular specialists, and any multidisciplinary clinicians (e.g., pediatricians, cardiologists, pulmonologists, orthopedists) and care teams involved in the transition of patients with SMA from pediatric to adult care. 

Learning Objectives

Upon completion of the educational activity, participants should be able to:

  • Recognize the evolving presentations of SMA in the treatment era and how they may change as patients age to adequately understand individual needs and establish care plans accordingly  
  • Discuss the role of FDA-approved SMA therapeutics, as well as their ongoing evaluation in broadening patient populations (e.g., adult, previously treated), in the transition from pediatric to adult care to better coordinate treatment continuity or make appropriate therapeutic adjustments   
  • Describe effective transition strategies for patients with SMA as they enter adulthood to ensure seamless, comprehensive, and continued care throughout their lifespans 
Course summary
Course opens: 
Course expires: 
Event starts: 
10/26/2023 - 6:00pm EDT
Event ends: 
10/26/2023 - 7:30pm EDT


Note: Each section will include group discussion topics and opportunities to troubleshoot your own practice challenges with other SMA treaters

The Current Picture of Pediatric to Adult Transitions in SMA

  • The changing natural history of SMA with current therapies
  • What happens when transitions go wrong
  • Importance and challenges of maintaining therapy into adulthood; proposed solutions by agent

The Pediatric Side of Transitions

  • Transition timeline for success
  • Key clinical tools and resources to streamline transition readiness and preparedness
  • Insight to potential barriers/challenges from both the pediatric and adult lens; proposed solutions

The Adult Side of Transitions

  • Key differences between pediatric and adult care
  • How to help a patient with SMA adapt to an adult care model
  • Navigating medical, logistical, and social obstacles

Registered attendees will have access to the Mütter Museum Exhibits for one hour at the conclusion of the presentation.

The Mütter Museum at The College of Physicians of Philadelphia
19 S 22nd St
Thomson Gallery, 1st Floor
Philadelphia, PA 19103
United States

Valet Parking has been arranged for registered attendees, please park in the lot behind the museum at 26 S 21st Street Philadelphia, PA 19103.


John Brandsema, MD
Child Neurologist
Neuromuscular Section Head
Children’s Hospital of Philadelphia
Philadelphia, PA



Lauren Elman, MD
Professor of Neurology
Perelman School of Medicine at the University of Pennsylvania
Philadelphia, PA

This activity is jointly provided by Medical Education Resources (MER) and Efficient LLC.


Accreditation Statement

In support of improving patient care, this activity has been planned and implemented by Medical Education Resources (MER) and Efficient LLC. MER is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

Designation Statement

Medical Education Resources designates this live activity for a maximum of 1.5 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Medical Education Resources has been authorized by the American Academy of Physician Assistants (AAPA) to award AAPA Category 1.5 CME credit for activities planned in accordance with AAPA CME Criteria. This activity is designated for 1.5 AAPA Category 1 CME Credits.  Physician Assistants should only claim credit commensurate with the extent of their participation. 
A Certificate of Participation will be given upon completion of the activity enabling participants to register their credit with the appropriate licensing boards or associations.

Disclosure of Relevant Financial Relationships

Medical Education Resources ensures balance, independence, objectivity, and scientific rigor in all our educational activities. In accordance with this policy, MER identifies relevant financial relationships with its instructors, content managers, and other individuals who are in a position to control the content of an activity. Reported relevant financial relationships are mitigated by MER to ensure that all scientific research referred to, reported, or used in a CE activity conforms to the generally accepted standards of experimental design, data collection, and analysis. MER is committed to providing learners with high-quality CE activities that promote improvements or quality in health care and not the business interest of an ineligible company.

Planners' and Managers' Disclosure

Efficient LLC and Medical Education Resources' planners and managers have no financial relationships to disclose.

Faculty Disclosure

Dr Brandsema reported the following financial relationships:

  • Consulting Fees: Argenx, AveXis/Novartis, Biogen, Fibrogen, Genentech, Momenta/Janssen, NS Pharma, Pfizer, PTC Therapeutics, Sarepta, Scholar Rock
  • Grants/Research Support: Alexion, Argenx, Astellas, AveXis/Novartis, Biogen, CSL Behring, Fibrogen, Genentech, Momenta/Janssen, Pfizer, PTC Therapeutics, Sarepta

Dr Elman reported the following financial relationships: 

  • Consulting Fees: Biogen, Genentech, Edgewise

Disclosure of Unlabeled Use/Disclaimer

This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications. The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.


The content and views presented in this educational activity are those of the authors and do not necessarily reflect those of Medical Education Resources, Efficient LLC, and/or Genentech. The authors have disclosed if there is any discussion of published and/or investigational uses of agents that are not indicated by the FDA in their presentations. Before prescribing any medicine, primary references and full prescribing information should be consulted. 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. The information presented in this activity is not meant to serve as a guideline for patient management.

Commercial Support

This continuing education activity is supported by an educational grant from Genentech, a member of the Roche group. 


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To attend in person, please select the "take course" button above.
5:30 – 6:00 PM Dinner & Registration 
6:00 – 7:30 PM Educational presentation 
7:30 – 8:30 PM The Mütter Museum will be available  to attendees for one hour after the conclusion of the presentation. 
To attend virtually via Zoom, please register here.
6:00 – 7:30 PM Educational presentation