Current and Emerging Clinical Trials in Mitochondrial Disease

Needs Statement

Clinical geneticists, residents, fellows, laboratory directors, genetic counselors, nurse practitioners, nurses, and medical and genetic counseling students need to receive regular updates on advances in clinical genetics research and in genomic diagnostics and therapeutics. Special emphasis will be given to fetal therapy for genetic disorders, therapeutic applications of the CRISPR/Cas9 system, and methods for decoding exome and genome sequences. The series aims to expand the knowledge of learners, provide the skills to interpret genomic studies, enhance the quality of medical genetics care, and improve patient outcomes.

Educational Objectives

At the conclusion of the activity, the participants should be able to:

• Recognize the potential therapeutic targets that could be used in mitochondrial disorders.
• Critique the results of existing clinical trials for mitochondrial disease.
• Analyze the potential efficacy of novel therapeutic interventions in mitochondrial disorders.

Target Audience

Activity Evaluation

Evaluation by questionnaire will address program content, presentation, and possible bias.

Educational Methods

• Lectures

Accreditation/Credit Designation

Baylor College of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Baylor College of Medicine designates this enduring material activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Social Work: The Baylor College of Medicine Division of Continuing Professional Development (License Number 7270) is an approved continuing education provider for Social Workers through the Texas State Board of Social Worker Examiners. This activity has been approved for 1 continuing education unit.

Term of Approval

September 1, 2024 through September 30, 2026. Original release date: September 1, 2024.

Disclosure Policy

Baylor College of Medicine (BCM) is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education (CME) for physicians. BCM is committed to sponsoring CE activities that are scientifically based, accurate, current, and objectively presented.

In accordance with the ACCME Standards for Commercial Support, BCM has implemented a mechanism requiring everyone in a position to control the content of an educational activity (i.e., directors, planning committee members, faculty) to disclose any relevant financial relationships with commercial interests (drug/device companies) and manage/resolve any conflicts of interest prior to the activity. Individuals must disclose to participants the existence or non-existence of financial relationships at the time of the activity or within 24 months prior.

In addition, BCM has requested activity faculty/presenters to disclose to participants any unlabeled use or investigational use of pharmaceutical/device products; to use scientific or generic names (not trade names) in referring to products; and, if necessary to use a trade name, to use the names of similar products or those within a class. Faculty/presenters have also been requested to adhere to the ACCME's validation of clinical content statements.

BCM does not view the existence of financial relationships with commercial interests as implying bias or decreasing the value of a presentation. It is up to participants to determine whether the relationships influence the activity faculty with regard to exposition or conclusions. If at any time during this activity you feel that there has been commercial/promotional bias, notify the Activity Director or Activity Coordinator. Please answer the questions about balance and objectivity in the activity evaluation candidly.

All of the relevant financial relationships listed for these individuals have been mitigated.