Rett Syndrome: Hope for Patients and Caregivers
Speakers: Paige Nues, Director, Family EmpowermentDavid Lieberman, MD, PhD, Child NeurologistCary Fu, MD, Assistant Professor, Pediatrics
Duration: 60 minutes
Presented on: August 18, 2022
Director, Family EmpowermentPaige Nues leads initiatives focused on building bridges between patient families, clinicians, scientists, and industry to stimulate participation and engagement in research activities and Centers of Excellence, and to foster patient-centered research development and design. She provides a safety net of accurate information through education programs that empower families, caregivers, and support teams, including organ donation for Rett research. Paige served on IRSF’s Board of Directors as a volunteer Regional Representative and is parent founder of Katie’s Clinic for Rett and Related Disorders at UCSF Benioff Children’s Hospitals. She turned her full energy and passion to solving the mysteries of Rett syndrome when her daughter, Katie, was diagnosed with Rett syndrome in 2003.
David Lieberman, MD, PhD
David Lieberman, MD, PhD
Child NeurologistDavid Lieberman, MD, PhD, is a child neurologist at Boston Children’s Hospital. Dr. Lieberman completed his medical and doctorate degrees in Neuroscience at Stanford University School of Medicine. He continued his training at Johns Hopkins University School of Medicine, completing an internship in Pediatrics and a residency in Pediatric Neurology. Dr. Lieberman remained at Johns Hopkins as faculty for a few years before moving back to the west coast to take a faculty position in the Neurosciences Department at UC San Diego. Dr. Lieberman moved to Boston in 2015 and now serves as the Director of the Comprehensive Rett Syndrome Program at Boston Children’s Hospital where he sees over 100 patients with Rett syndrome and Rett-related disorders each year.
Cary Fu, MD
Cary Fu, MD
Assistant Professor, PediatricsCary Fu, MD, is an Assistant Professor of Pediatrics in the Division of Pediatric Neurology at Vanderbilt Medical Center and Medical Director of the Rett syndrome clinic at Vanderbilt Children’s Hospital. Originally from St. Louis, Missouri, Dr. Fu received his undergraduate and medical degrees from University of Missouri – Columbia. His clinical emphasis is in pediatric epilepsy and, academically, he has actively been involved in Rett syndrome related clinical and translational research for the past decade serving as principal investigator or co-investigator on several observational studies and clinical trials. Dr. Fu is board certified by the American Board of Psychiatry and Neurology and an active member in both the Child Neurology Society and American Epilepsy Society.
Rett Syndrome: Hope for Patients and Caregivers
ACKNOWLEDGEMENTThis activity is supported by an educational grant from Acadia Pharmaceuticals Inc.
DATE OF RELEASE/EXPIRATIONThis activity was released on August 18, 2022 and is valid until September 14, 2023. Requests for credit must be made no later than September 14, 2023.
TARGET AUDIENCEThe educational design of this activity addresses the needs of neurologists, pediatric neurologists, and neuromuscular specialists involved in the treatment of patients with Rett syndrome.
STATEMENT OF NEED/PROGRAM OVERVIEWRett syndrome is a rare, non-inherited neurodevelopmental disorder with no disease-specific therapies or cure. Despite this burden on patients and their caregivers, diagnosis is often delayed by 12 months or more after symptom onset. A call to action to clinicians for earlier diagnosis is urgently needed. Additionally, a variety of emerging therapies hold great promise for improving the treatment of Rett syndrome, and warrants education for clinicians to enhance awareness of new and effective treatment options to manage symptoms.
Join a panel of expert neurologists for an exploration of the urgent need for earlier diagnoses of Rett syndrome to ensure proper management. Panelists will review the stages of Rett syndrome, the criteria for diagnosis, the genetic mutations that cause the disease, and the place for genetic testing. Participating clinicians will recognize the available options to manage symptoms (gastrointestinal dysfunction, epilepsy, and physical and language deficits) and the promising therapies being investigated in clinical trials.
EDUCATIONAL OBJECTIVESAfter completing this activity, the participant should be better able to:
- Recognize signs and symptoms of Rett syndrome early in its disease course
- Discuss the place of genetic testing in the diagnosis of Rett syndrome
- Summarize the components of the symptomatic management of patients with Rett syndrome
- Review the therapeutic potential of agents being investigated for the treatment of patients with Rett syndrome
PHYSICIAN ACCREDITATION STATEMENTThis 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 Global Education Group (Global) and PlatformQ Health Education, LLC. Global is accredited by the ACCME to provide continuing medical education for physicians.
PHYSICIAN CREDIT DESIGNATIONGlobal Education Group 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.
ESTIMATED TIME TO COMPLETEThis activity should take approximately 60 minutes to complete.
INSTRUCTIONS TO RECEIVE CREDITIn order to receive credit for this activity, the participant must (1) read the target audience, learning objectives, and disclosure statements, (2) complete the educational activity online, and (3) complete the post-test and activity evaluation. To receive AMA PRA Category 1 Credits™, participants must receive a score of 70% on the post-test.
HARDWARE/SOFTWARE REQUIREMENTSParticipants will need a computer with a recent version of Adobe Flash installed, as well as an internet connection sufficient for streaming media.
FEE INFORMATION & REFUND/CANCELLATION POLICYThere is no fee for this educational activity.
DISCLOSURE OF CONFLICTS OF INTERESTGlobal Education Group (Global) adheres to the policies and guidelines, including the Standards for Integrity and Independence in Accredited CE, set forth to providers by the Accreditation Council for Continuing Medical Education (ACCME) and all other professional organizations, as applicable, stating those activities where continuing education credits are awarded must be balanced, independent, objective, and scientifically rigorous. All persons in a position to control the content of an accredited continuing education program provided by Global are required to disclose all financial relationships with any ineligible company within the past 24 months to Global. All financial relationships reported are identified as relevant and mitigated by Global in accordance with the Standards for Integrity and Independence in Accredited CE in advance of delivery of the activity to learners. The content of this activity was vetted by Global to assure objectivity and that the activity is free of commercial bias.
All relevant financial relationships have been mitigated.
The faculty have the following relevant financial relationships with ineligible companies:
Name of Faculty or Presenter Reported Financial Relationship Cary Fu, MD
Child Neurologist and Epileptologist
Medical Director, Vanderbilt Rett Syndrome Clinic
Vanderbilt University Medical Center
Department of Pediatrics
Nothing to disclose David N. Lieberman, MD, PhD
Director, Comprehensive Rett Syndrome Clinic
Department of Neurology
Boston Children’s Hospital
Consulting Fee: (e.g., Advisory Board): Acadia Pharmaceuticals, Neurogene, and Taysha Gene Therapies
Contracted Research: PI for clinical trials supported by Acadia Pharmaceuticals, Anavex Life Sciences, and GW Research Limited
Director of Family Empowerment
International Rett Syndrome Foundation (IRSF)
Nothing to disclose
The planners and managers have the following relevant financial relationships with ineligible companies:
Name of Planner or Manager Reported Financial Relationship Lindsay Borvansky Nothing to disclose Andrea Funk Nothing to disclose Liddy Knight Nothing to disclose Ashley Cann Nothing to disclose Carole Drexel, PhD Nothing to disclose Colleen Heffner Nothing to disclose Jaimee Harris-Gold Nothing to disclose Stephanie Puccio Nothing to disclose Samantha Brant – Caregiver Participant Nothing to disclose
DISCLOSURE OF UNLABELED USEThis educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. Global Education Group (Global) and PlatformQ Health Education, LLC 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 any organization associated with this activity. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.
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 in this activity should not be used by clinicians without evaluation of patient conditions and possible contraindications on dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.
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