Clinical Consultations™: Expert Perspectives in Choroideremia – Analyzing the Potential Impact of Gene Therapy

Clinical Consultations™: Expert Perspectives in Choroideremia – Analyzing the Potential Impact of Gene Therapy

M. Dominik Fischer, MD, PhD, FEBO; Byron L. Lam, MDfg

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Activity Overview

Choroideremia (CHM) is a rare, X-linked, inherited retinal disorder characterized by progressive vision loss, usually beginning with night-blindness, followed by peripheral vision loss, and eventually leading to complete loss of sight. The disease is associated with mutations in the CHM gene that lead to degeneration of the photoreceptors, retinal pigment epithelium, and choroid. Although there is no cure, advances in CHM pathogenetics have had significant clinical implications. Clinical gene therapy trials in patients with CHM have demonstrated the potential for slowing or even stopping the degeneration characteristic of this disease. As CHM gene therapy approaches regulatory approval, clinicians need to remain up-to-date with these developments so they can recommend appropriate treatments as they become available.
In this on-demand, online CME activity, two faculty experts in choroideremia and gene therapy discuss the pathophysiology, diagnosis, and emerging treatments for CHM. In their discussion, they share their experiences in caring for patients with choroideremia, giving learners practical insights into this rare disease. Their firsthand experience with clinical trials sheds new light on the potential role of emerging gene therapies in the treatment of this disease.

Acknowledgement of Commercial Support

This activity is supported by an educational grant from Biogen.

Target Audience

This online educational activity is directed toward retinal specialists and ophthalmologists. Other healthcare providers involved of the care of patients suffering from CHM are also invited to participate.

Learning Objectives

    Upon successful completion of this activity, you should be better prepared to:
  • Discuss the role of the CHM gene in the pathophysiology of choroideremia
  • Identify the clinical features of choroideremia that differentiate it from similar retinal diseases
  • Evaluate the clinical trial data for current and emerging gene therapies in the treatment of choroideremia

Faculty Staff and Planners' Disclosures

The staff of Physicians’ Education Resource®, LLC have no relevant financial relationships with ineligible entities.

Faculty

M. Dominik Fischer, MD, PhD, FEBO M. Dominik Fischer, MD, PhD, FEBO
Professor of Ophthalmology
Oxford Eye Hospital
Oxford University Hospitals NHS Foundation Trust
Oxford, UK
Disclosures: Grant Research Support: Biogen, Novartis; Consultant: Biogen, Novartis, F. Hoffman-La Roche, Janssen, REGENXBIO, SIRION Biotech, ARCTOS Medical Technologies, Frontera Therapeutics; Other: Patent holder (50%) for treatment of retinitis pigmentosa.

Byron L. Lam, MD Byron L. Lam, MD
Professor of Ophthalmology
Robert Z. & Nancy J. Greene Chair in Ophthalmology
Bascom Palmer Eye Institute
University of Miami Miller School of Medicine
Miami, FL
Disclosures: Grant Research Support: Biogen; Consultant: Biogen.

PER® mitigated all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.

Off-Label Disclosure and Disclaimer

This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a healthcare professional relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER® or any company that provided commercial support for this activity.

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Medical Crossfire® Runs: Mar 22nd 2021 – Mar 22nd 2022

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Accreditation/Credit Designation

Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Physicians’ Education Resource®, LLC, 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.

Acknowledgment of Support

This activity is supported by an educational grant from ____.