After the Honeymoon is Over:

Side effects, late effects, and long-term challenges of gene therapy in neurologic disease

Presented by

Michael Hendricks, MD

Assistant Professor of Neurology

Division of Child Neurology

University of Kentucky

Wednesday, August 5, 2026

8:00 - 9:00 AM EDT

Zoom only: https://uky.zoom.us/j/89948846670

After this session, participants will be able to:

1. Describe the major acute and long-term adverse effects of gene therapies used for neurologic disorders, with emphasis on SMA and Duchenne muscular dystrophy.
2. Evaluate the major challenges that emerge after gene therapy, including immune-mediated toxicity, durability of benefit, inability to re-dose, and delayed treatment-related complications.
3. Apply lessons from current gene therapies to the clinical counseling and long-term monitoring of patients and to the evaluation of emerging gene-therapy approaches for neurologic disease.

Disclosure of Financial Interest Summary

All planners, faculty, and others in control of educational content are required to disclose all their financial relationships with ineligible companies within the prior 24 months. An ineligible company is defined as one whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients. Financial relationships are relevant if the educational content an individual can control is related to the business lines or products of the ineligible company.

None of the planners, faculty, and others in control of educational content for this educational activity have a relevant financial relationship(s) to disclose with ineligible companies.

The material presented in this course represents information obtained from the scientific literature as well as the clinical experiences of the speakers. In some cases, the presentations might include a discussion of investigational agents and/or off-label indications for various agents used in clinical practice. Speakers will inform the audience when they are discussing investigational and/or off-label uses.

Disclosure of a relationship is not intended to suggest or condone commercial bias in any presentation, but it is made to provide participants with information that might be of potential importance to their evaluation of a presentation.