Activity Overview
This online, on-demand virtual symposium brings together renowned experts in thymidine kinase 2 deficiency (TK2d). In this educational program, these experts discuss the pathophysiology of TK2d, as well as the importance of timely diagnosis and evidence-based treatment approaches. Additionally, they share their perspectives and best practices and forecast applications of key clinical challenges and questions regarding identification of this rare disease.
This educational activity is an archive of the live virtual symposium held on October 16, 2026.
Target Audience
This educational activity is directed toward neurologists, neuromuscular specialists, pediatric neurologists, PAs, RNs, and other healthcare professionals involved in the care of patients with T2Kd.
Learning Objectives
Upon successful completion of this activity, you should be better prepared to:
- Outline the relationship between the pathophysiological pathways underlying TK2d and its clinical presentations
- Describe diagnostic strategies to identify patients with TK2d
- Identify the limitations of current treatment paradigms for patients with TK2d
- Analyze clinical trial data for agents for TK2d
Michio Hirano, MD
Lucy G. Moses Professor of Neurology
Chief, Neuromuscular Medicine Division
Director, H. Houston Merritt Neuromuscular Research Center
Columbia University Irving Medical Center
New York, NY
Disclosures: Advisor, Consultant, Fee for Service Recipient: Cellergy, Golden Heart Flowers, UCB; Speakers Bureau: Apollo Medical Communications, Meducom, MJH Life Sciences; Grant/Research Funding: Biogen, Cyclerion, Department of Defense, MDA, Mighty Therapeutics (formerly Stealth BioTherapeutics), NIH, Pharming/Abliva Therapeutics, POLG Foundation, Reneo, Sarepta, Tisento Therapeutics; Royalty or Patent Benefits: Received Royalties as co-inventor in patent on nucleoside therapy for mitochondrial DNA depletion/deletions syndrome.
Mary Kay Koenig, MD
Professor & Associate Vice-Chair for Clinical Research
Endowed Chair of Mitochondrial Medicine
Director, Pediatric & Neurologic Clinical Research Unit
Director, Center for the Treatment of Pediatric Neurologic & Neurodegenerative Disease
Co-Director, Tuberous Sclerosis Center
Department of Pediatrics, Division of Child and Adolescent Neurology
McGovern Medical School at UTHealth Houston
Houston, TX
Disclosures: Advisor, Consultant, Fee for Service Recipient: ChromaDex, Jazz Pharma, Mighty/Stealth Thera, Pretzel Thera, Saol, Tisento Thera, UCB; Speakers Bureau: Jazz Pharmaceuticals; Grant/Research Funding: Astellas Pharm, Eli Lilly, GRIN Thera, Harmony Bioscience, Insmed Inc., Jazz Pharm, Khondrion BV, Leigh Syndrome International Consortium, Longboard Pharm, Pfizer, Pharming, Praxis, PTC Thera, Quince Thera, Supernus Pharm, Tisento Thera, UCB, United Mitochondrial Disease Foundation, Xenon Pharma; Royalty or Patent Benefits: LAM Therapeutics.
Zarazuela Zolkipli-Cunningham, MBChB, MRCP
Clinical Research Director and Attending Physician
Mitochondrial Medicine Frontier Program
Division of Human Genetics
The Children’s Hospital of Philadelphia
Assistant Professor of Pediatrics
University of Pennsylvania
Philadelphia, PA
Disclosures: Zarazuela Zolkipli-Cunningham has no relevant financial relationships with ineligible companies.
The staff of Physicians’ Education Resource®, LLC, have no relevant financial relationships with ineligible companies.
PER® mitigated all conflicts of interest for faculty, staff, and planners prior to the start of this activity by using a multistep process.
Off-Label Disclosure and Disclaimer
This accredited continuing education activity was planned in accordance with the ACCME Standards for Integrity and Independence to ensure balance, objectivity, independence, and scientific rigor. Faculty were instructed to use generic names when possible and to base recommendations on the best available evidence. Proprietary product names may be referenced in clinical discussion for educational purposes only and do not imply endorsement.
This activity may include discussion of investigational, unapproved, or off‑label uses of drugs or devices. Learners are advised to consult prescribing information for products discussed. Content is provided for educational purposes only and is not a substitute for the independent clinical judgment of a health care professional. Learners are encouraged to critically evaluate the information presented and apply their own clinical judgment.
The views expressed are those of the individual faculty members and do not necessarily reflect the views of the accredited provider or any entity providing commercial support.
This activity may have used AI-assisted tools to support educational development. All content was reviewed and approved by qualified faculty or planners. The accredited provider retains full responsibility for accuracy, balance, and independence.
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