In this episode, David N. Lieberman, MD, PhD, and Eric Marsh, MD, PhD, discuss recent updates relevant to the treatment of Rett syndrome, including:

• The recently updated Comprehensive Care Guidelines from the International Rett Syndrome Foundation, available at rettsyndrome.org
• Ongoing collection and analysis of longitudinal data within the Rett Syndrome Natural History Study
• Efficacy and safety data from the double-blind, placebo-controlled phase III registrational LAVENDER trial evaluating oral trofinetide, a small molecule analogue of insulin-like growth factor 1
• Topline results from phase III AVATAR and EXCELLENCE trials of the sigma-1 receptor agonist blarcamesine
• Several early ongoing gene therapy trials

Dr Lieberman and Dr Marsh complement this informative update with wisdom from their own clinical experience to provide a full picture of current treatment and management options available to patients with Rett syndrome.

Presenters:

David N. Lieberman, MD, PhD
Director, Comprehensive Rett Syndrome Clinic
Department of Neurology
Boston Children's Hospital
Boston, Massachusetts

Eric Marsh, MD PhD
Clinical Director, Orphan Disease Center
Director (MPI), CHOP/Penn IDDRC
Professor, Departments of Neurology and Pediatrics
Division of Neurology
Children’s Hospital of Philadelphia
Perelman School of Medicine
University of Pennsylvania
Philadelphia, Pennsylvania

This content is part of an online CME program in partnership with the International Rett Syndrome Foundation and supported by an educational grant from Acadia Pharmaceuticals. Link to full program:
https://bit.ly/4fJRRL0

Link to CME: Claim Credit - https://bit.ly/3OByCaE

In this episode, Drs. Halford and Hahn discuss the current treatment landscape of developmental and epileptic encephalopathies (DEEs), such as Lennox-Gastaut syndrome and Dravet syndrome, and the emerging role of brain cholesterol in addressing seizure frequency. The full episode covers:

• How to diagnose these epilepsies and the FDA-approved treatment options in the first-line and second-line settings
• The role of antiseizure medications in DEE treatment management
• The nonpharmacological treatment options available to patients with DEEs
• The emerging role of brain cholesterol in neurologic diseases like DEEs and Alzheimer’s disease
• Results from the ELEKTRA clinical trial, including cholesterol synthase inhibitors’ impact on seizure frequency
• The future of soticlestat as a treatment option for pediatric patients with DEEs

Presenters:

Jonathan J. Halford, MD
Professor
Director, Translational Research Unit
Department of Neurology
Medical University of South Carolina
Charleston, SC

Cecil Hahn, MD, MPH, FRCPC
Professor
Pediatric Epileptologist
University of Toronto
Toronto, Ontario, Canada

Content based on an online CME program supported by an independent educational grant from Takeda Pharmaceuticals U.S.A., Inc.

Link to full program: 
https://bit.ly/45UUpQy

Link to CME: Claim Credit - https://bit.ly/3wbVkA1

In this episode, Drs. Halford and White discuss the emerging role of brain cholesterol in the development and potential treatment of neurological diseases like epilepsy or Alzheimer and Huntington diseases. The full episode covers:

• Overview of brain cholesterol and its function in the body
• Pathogenesis potential for certain diseases via brain cholesterol
• Emerging research on a cholesterol synthase inhibitor as a novel therapy
• The future of soticlestat as a potential treatment option for pediatric epilepsies—Lennox-Gastaut syndrome and Dravet syndrome—and other neurological diseases

Presenters:

Jonathan J. Halford, MD
Professor
Director, Translational Research Unit
Department of Neurology
Medical University of South Carolina
Charleston, SC

H. Steve White, PhD
Professor, Department of Pharmacy
Co-Director, Center for Epilepsy Drug Discovery
School of Pharmacy
University of Washington
Seattle, Washington

In this episode, Drs. Sabbagh and Isaacson discuss how to best approach Alzheimer’s disease (AD) and mild cognitive impairment (MCI) in clinical practice, including accurately diagnosing patients and optimizing treatment in terms of patient preferences and reimbursement options. The full episode covers:

• Defining AD and MCI and their clinical presentation
• How to diagnose AD vs MCI
• Reimbursement landscape for current diagnosing and monitoring tools
• Precision medicine’s evolving role in diagnosing and treating patients with AD and/or MCI

Presenters:

Marwan Sabbagh, MD
Professor of Neurology
Alzheimer’s and Memory Disorders Division
Department of Neurology
Barrow Neurological Institute
Phoenix, Arizona

Richard Isaacson MD
Director of the Institute for Neurodegenerative Diseases
Florida

The treatment landscape of major depression is increasingly shifting from monoaminergic agents to agents that modulate glutamate and GABA. Healthcare professionals need to stay up to date and educated on these changes in order to discuss the most current treatment options with their patients. In this podcast episode, we interview Dr Anita Clayton, a clinician researcher who has been involved in some of the clinical trials for these novel glutamatergic and GABAergic medications, and ask her key questions about mechanism of action and clinical application. Listeners will come away from this podcast episode more informed and more excited for the future of major depression care. 

Presenters:

Anita Clayton, MD
David C. Wilson Professor and Chair
Department of Psychiatry and Neurobehavioral Sciences
Professor of Clinical Obstetrics and Gynecology
University of Virginia School of Medicine
Charlottesville, Virginia

This podcast episode is supported by an educational grant from Sage Therapeutics, Inc. and Biogen.

Link to full program:
https://bit.ly/3YdPMPa

Rett syndrome is a rare but severe neurodevelopmental disorder, and current treatment approaches primarily focus on symptom management and supportive care. However, recent clinical developments are providing hope for a changing treatment landscape. In this podcast episode, experts in Rett syndrome David N. Lieberman, MD, PhD, and Jeffrey Neul, MD, PhD, discuss trofinetide and blarcamesine, 2 agents in late-stage clinical trials as treatment options for Rett syndrome. Throughout their conversation, Drs Lieberman and Neul review the pivotal LAVENDER, DAFFODIL, AVATAR, and EXCELLENCE trials, including information on the safety and efficacy profiles of trofinetide and blarcamesine. The experts end their discussion with a conversation about the potential roles of ketamine and gene therapy in the management of Rett syndrome.      

Presenters:

David N. Lieberman, MD, PhD
Instructor
Department of Neurology
Harvard Medical School
Attending Physician
Child Neurology
Boston Children’s Hospital
Boston, Massachusetts

Jeffrey Neul, MD, PhD
Director
Pediatrics
Vanderbilt University Medical Center
Nashville, Tennessee

This activity is supported by educational funding provided by Acadia Pharmaceuticals Inc.

To claim CME/CE credit for this podcast, complete the posttest here:
 bit.ly/3JR4qq5.

For additional activities in this program, visit
 bit.ly/3JR4qq5

Rett syndrome affects many organ systems, and each patient has individual symptoms and experiences—there is no “one-size-fits-all” treatment approach. In this podcast episode, Rett syndrome experts David N. Lieberman, MD, PhD, and Shannon Standridge, DO, MPH, discuss some of the more common manifestations of Rett syndrome and current management strategies. While covering manifestations such as epilepsy, behavioral issues, and gastrointestinal distress, they highlight the value of a team approach to help the patient thrive. Throughout their conversation, Drs Lieberman and Standridge weave in real-world examples of interactions they have had with families of patients with Rett syndrome and offer insights on communication strategies for different aspects of management.

Presenters:

David N. Lieberman MD, PhD
Instructor
Department of Neurology
Harvard Medical School
Attending Physician
Child Neurology
Boston Children’s Hospital
Boston, Massachusetts

Shannon Standridge, DO, MPH
Professor
Division of Neurology
Vinaya Rett Syndrome and Related Spectrum Disorders Clinic
Cincinnati Children’s Hospital Medical Center
Cincinnati, Ohio

This activity is supported by educational funding provided by Acadia Pharmaceuticals Inc.

To claim CME/CE credit for this podcast, complete the posttest here: 
http://bit.ly/41CeaeP

For additional activities in this program, visit 
http://bit.ly/3lXuzda

In this podcast episode, Leslie Citrome, MD, MPH, and Adam Lowy, MD, share a wealth of knowledge about long-acting injectable (LAI) antipsychotics. Their conversation begins with an overview of the pharmacokinetic profiles of LAI antipsychotics and how they differ from their oral counterparts, as well as injection intervals available with current formulations. The experts also discuss the clinical utility of LAI antipsychotics in psychiatry practice, highlighting their role in assuring medication adherence and their application in both first- and later-episode psychosis. Finally, Drs Citrome and Lowy hit on practical aspects of incorporating LAI antipsychotics into patient care: communicating with patients to dispel myths and stigma surrounding injectable medications, identifying injection resources in your medical community, and strategies to navigate insurance barriers.

Throughout the podcast, Drs Citrome and Lowy reference several links to outside sources that can provide valuable education on LAI antipsychotics. Those links are listed below for your convenience.

Administering LAI Into Dorsal Gluteal Site: 
https://bit.ly/3XjSm5a

Administering LAI Into Deltoid Site: 
https://bit.ly/3Xlyq1L

Administering LAI Into Abdominal Site: 
https://bit.ly/3wgGJ2Z

Presenters:

Leslie Citrome, MD, MPH
Clinical Professor
Department of Psychiatry and Behavioral Sciences
New York Medical College
Valhalla, New York

Adam Lowy, MD
Staff Psychiatrist
Ellenhorn
Los Angeles, California

This activity is supported by educational funding provided byOtsuka America Pharmaceutical, Inc. and Lundbeck.

For additional activities in this program, visit:
https://bit.ly/3iM0HiP