Release Date: October 31, 2025
Expiration Date: October 31, 2026
Activity Overview
This video-based program is designed for health care professionals to explore recent advances in the management of patients with thymidine kinase 2 deficiency (TK2d). This online activity focuses on the pathophysiology, diagnosis, and management of TK2d. Further, data regarding investigational agents that may be game changers for the treatment of this disease are reviewed. The expert faculty discuss many of the issues facing clinicians who care for patients with TK2d.
Target Audience
These educational activities are directed toward pediatric neurologists, neurologists, genetic counselors, specialty NPs/PAs, RNs, pediatricians, and other HCPs who may encounter TK2d.
Learning Objectives
Upon completion of this educational activity, learners should be able to:
- Describe the relationship between the pathophysiological pathways underlying TK2d and its clinical presentations
- Implement diagnostic strategies to identify patients with TK2d
- Review the limitations of current treatment paradigms for patients with TK2d
- Evaluate clinical trial data for agents for TK2d
Zarazuela Zolkipli-Cunningham, MBChB, MRCP
Assistant
Professor of Pediatrics, Perelman School of Medicine
University
of Pennsylvania
Attending
Physician and Clinical Research Director
Mitochondrial
Medicine Frontier Program (MMFP)
Division
of Human Genetics, Children’s Hospital of Philadelphia
Philadelphia,
PA
Disclosures: Advisor,
Consultant, Speaker, Honoraria Recipient: Adjudvia Therapeutics, Astellas
[formerly Mitobridge] Pharma Inc., BPGBio, Imel Therapeutics, Khondrion,
National Institutes of Health, North American Mitochondrial Disease Consortium,
Pediatric Neuromuscular Research Network Consortium, Precision Therapeutics,
Pretzel Therapeutics, Reneo Therapeutics, Ribonova Inc., Stealth
BioTherapeutics, Thiogenesis Therapeutics, Tisento Therapeutics, UCB Pharma,
United Mitochondrial Disease Foundation, US Department of Defense
Mary Kay Koenig, MD
Professor of Pediatrics
Director, Mitochondrial Center of Excellence
Director, Center for the Treatment of Pediatric Neurodegenerative
Disease
University of Texas McGovern Medical School, UTHealth
Houston, TX
Disclosures: Advisor,
Consultant, Speaker, Honoraria Recipient: Acasti
Pharma, Astellas Pharma Inc, Chromadex, Jazz Pharmaceuticals, Marinus
Pharmaceuticals, Quince Therapeutics, Tisento Therapeutics; Grant/Funding
Research: Abliva AB, Astellas Pharma, Emalex Biosciences Inc., GRIN
Therapeutics, GW Research Ltd, Jazz Pharmaceuticals, Longboard, Marinus
Pharmaceuticals, Noema Pharma, Pfizer, PTC Therapeutics, Quince Therapeutics,
Stealth BioTherapeutics, Tisento Therapeutics, UCB, Xenon Pharma
Perry Shieh, MD, PhD, FAAN, FANA
Professor of Neurology and Pediatrics
Ronald Reagan UCLA Medical Center
David Geffen School of Medicine at UCLA
University of California Los Angeles
Los Angeles, CA
Disclosures: Advisor, Consultant, Speaker,
Honoraria Recipient: Alexion, Argenx, Biogen, Catalyst, CSL Behring,
Grifols, Pfizer, Sarepta, Scholar Rock; Grant/Research Funding: Abcuro,
Arcellx, Astellas, Avidity, Biogen, Dyne, Novartis, Pfizer, Sarepta, Solid, UCB
Faculty, Staff, and Planners’ Disclosures
The staff of Physicians’ Education Resource®, LLC have no relevant financial relationships with ineligible companies.
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 health care 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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