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UCB presents latest thymidine kinase 2 deficiency (TK2d) research at the United Mitochondrial Disease Foundation (UMDF) 2025 Conference

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UCB announced its participation in the UMDF Mitochondrial Medicine 2025 Conference, presenting three key research posters on thymidine kinase 2 deficiency (TK2d). The presentations include findings from disease course studies in untreated patients, examining progression and outcomes over time, as well as insights into caregiver burden from the Assessment of TK2d Patient Perspectives study. The research focuses on both pediatric (≤12 years) and adult (>12 years) onset patients, representing data from the largest international TK2d dataset. Dr. Kimberly A. Moran, SVP & Head of US Rare Diseases at UCB, emphasized the company's commitment to advancing understanding of this rare mitochondrial disease, which has historically lacked research attention.
UCB ha annunciato la sua partecipazione alla conferenza UMDF Mitochondrial Medicine 2025, presentando tre poster di ricerca chiave sulla carenza di timidina chinasi 2 (TK2d). Le presentazioni includono risultati di studi sul decorso della malattia in pazienti non trattati, analizzando la progressione e gli esiti nel tempo, oltre a informazioni sul carico dei caregiver derivanti dallo studio Assessment of TK2d Patient Perspectives. La ricerca si concentra sia su pazienti pediatrici (≤12 anni) sia adulti (>12 anni), rappresentando dati del più grande database internazionale TK2d. La dottoressa Kimberly A. Moran, SVP e Responsabile delle Malattie Rare USA presso UCB, ha sottolineato l'impegno dell'azienda nell'avanzare la comprensione di questa rara malattia mitocondriale, storicamente trascurata dalla ricerca.
UCB anunció su participación en la conferencia UMDF Mitochondrial Medicine 2025, presentando tres pósteres de investigación clave sobre la deficiencia de timidina quinasa 2 (TK2d). Las presentaciones incluyen hallazgos de estudios sobre la evolución de la enfermedad en pacientes no tratados, examinando la progresión y los resultados a lo largo del tiempo, así como perspectivas sobre la carga de los cuidadores del estudio Assessment of TK2d Patient Perspectives. La investigación se centra en pacientes con inicio pediátrico (≤12 años) y adulto (>12 años), representando datos del mayor conjunto internacional de datos TK2d. La Dra. Kimberly A. Moran, SVP y Jefa de Enfermedades Raras en EE. UU. de UCB, destacó el compromiso de la compañía para avanzar en la comprensión de esta rara enfermedad mitocondrial, que históricamente ha carecido de atención investigativa.
UCB는 UMDF 미토콘드리아 의학 2025 컨퍼런스에 참가하여 티미딘 키나제 2 결핍증(TK2d)에 관한 세 가지 주요 연구 포스터를 발표했습니다. 발표 내용에는 치료받지 않은 환자들의 질병 경과 연구 결과, 시간에 따른 진행 및 결과 분석, 그리고 TK2d 환자 관점 평가 연구에서 도출된 돌봄 제공자의 부담에 대한 인사이트가 포함되어 있습니다. 연구는 소아(≤12세) 및 성인(>12세) 발병 환자 모두를 대상으로 하며, 가장 큰 국제 TK2d 데이터셋의 데이터를 반영합니다. UCB 미국 희귀질환 담당 수석 부사장 겸 책임자인 Dr. Kimberly A. Moran은 이 희귀 미토콘드리아 질환에 대한 이해를 증진시키려는 회사의 의지를 강조했습니다. 이 질환은 그동안 연구가 부족했던 분야입니다.
UCB a annoncé sa participation à la conférence UMDF Mitochondrial Medicine 2025, présentant trois posters de recherche clés sur la déficience en thymidine kinase 2 (TK2d). Les présentations incluent des résultats d'études sur l'évolution de la maladie chez des patients non traités, examinant la progression et les résultats au fil du temps, ainsi que des perspectives sur la charge des aidants issues de l'étude Assessment of TK2d Patient Perspectives. La recherche porte sur des patients à début pédiatrique (≤12 ans) et adulte (>12 ans), représentant les données du plus grand ensemble international TK2d. La Dre Kimberly A. Moran, SVP et responsable des maladies rares aux États-Unis chez UCB, a souligné l'engagement de l'entreprise à faire progresser la compréhension de cette maladie mitochondriale rare, historiquement peu étudiée.
UCB kündigte seine Teilnahme an der UMDF Mitochondrial Medicine 2025 Konferenz an und präsentierte drei wichtige Forschungsplakate zur Thymidin-Kinase-2-Mangel (TK2d). Die Präsentationen umfassen Ergebnisse von Studien zum Krankheitsverlauf bei unbehandelten Patienten, die den Fortschritt und die Ergebnisse im Zeitverlauf untersuchen, sowie Einblicke in die Belastung der Pflegepersonen aus der Studie Assessment of TK2d Patient Perspectives. Die Forschung konzentriert sich auf Patienten mit pädiatrischem (≤12 Jahre) und erwachsenem (>12 Jahre) Beginn und basiert auf den Daten des größten internationalen TK2d-Datensatzes. Dr. Kimberly A. Moran, SVP und Leiterin der US-Abteilung für Seltene Krankheiten bei UCB, betonte das Engagement des Unternehmens, das Verständnis dieser seltenen mitochondrialen Erkrankung voranzutreiben, die bisher wenig Forschungsaufmerksamkeit erhalten hat.
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  • UCB will contribute three presentations, including data on the disease course of thymidine kinase 2 deficiency (TK2d) in untreated patients and insights into caregiver impact
  • These data emphasize UCB's commitment to advancing treatment outcomes and patient experiences in TK2d and uncovering important insights into this rare disease

ATLANTA, June 20, 2025 /PRNewswire/ -- UCB, a global biopharmaceutical company, announced it will present pioneering TK2d research at the UMDF Mitochondrial Medicine 2025 Conference taking place June 18-21, 2025, in St. Louis, Missouri.

UCB will present findings from disease course studies of TK2d in untreated patients, looking at disease progression and outcomes over time,1,2 as well as real-world insights into the burden and impact on caregivers, providing a comprehensive view of the experiences and challenges faced by those supporting individuals with TK2d.3

"Our participation in the UMDF 2025 Conference underscores our commitment to advancing the understanding of TK2d, an area previously lacking in research," said Kimberly A. Moran, PhD, MBA, SVP & Head, US Rare Diseases. "We are excited to introduce new findings that have the potential to impact people living with this rare mitochondrial disease."

UCB presentations at UMDF 2025

Lead author

 

Title

Presentation details

M Hirano et al.

The Disease Course of Untreated Patients with Thymidine
Kinase 2 Deficiency (TK2d) Aged ≤12 Years at TK2d
Symptom Onset: Findings from the Largest International
TK2d Dataset

Poster

M Hirano et al.

The Disease Course of Untreated Patients with Thymidine
Kinase 2 Deficiency (TK2d) Aged >12 Years at TK2d
Symptom Onset: Findings from the Largest International
TK2d Dataset

Poster

A Karaa et al.

Burden and impact of caring for those with thymidine
kinase 2 deficiency (TK2d): results from the Assessment of
TK2d Patient Perspectives (ATP) study

Poster

For further information, contact UCB:

Rare Disease Communications
Daphne Teo
T +1 (770) 880-7655
daphne.teo@ucb.com  

Global Communications
Nick Francis
T +44 7769 307745
nick.francis@ucb.com 

Corporate Communications, Media Relations
Laurent Schots
T +32.2.559.92.64
laurent.schots@ucb.com

Investor Relations
Antje Witte   
T +32.2.559.94.14
antje.witte@ucb.com

About UCB
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9000 people in approximately 40 countries, the company generated revenue of € 6.15 billion in 2024. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news.

Forward-looking statements
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UCB is providing this information, including forward-looking statements, only as of the date of this press release and it does not reflect any potential impact from the evolving COVID-19 pandemic, unless indicated otherwise. UCB is following the worldwide developments diligently to assess the financial significance of this pandemic to UCB. UCB expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations.

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References:

  1. Hirano, M., et al. The Disease Course of Untreated Patients with Thymidine Kinase 2 Deficiency (TK2d) Aged ≤12 Years at TK2d Symptom Onset: Findings from the Largest International TK2d Dataset.
  2. Hirano, M., et al. The Disease Course of Untreated Patients with Thymidine Kinase 2 Deficiency (TK2d) Aged >12 Years at TK2d Symptom Onset: Findings from the Largest International TK2d Dataset.
  3. Karaa, A., et al. Burden and impact of caring for those with thymidine kinase 2 deficiency (TK2d): results from the Assessment of TK2d Patient Perspectives (ATP) study.

©2025 UCB, Inc., Smyrna, GA 30080. All rights reserved. US-MT-2500121

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SOURCE UCB

FAQ

What research is UCB presenting at the UMDF 2025 Conference?

UCB is presenting three posters: two on disease course studies in untreated TK2d patients (for ages ≤12 and >12 years), and one on caregiver burden from the ATP study.

When and where is the UMDF Mitochondrial Medicine 2025 Conference taking place?

The conference is taking place from June 18-21, 2025, in St. Louis, Missouri.

What is the significance of UCB's TK2d research presentations?

The research represents findings from the largest international TK2d dataset, providing important insights into disease progression and caregiver experiences in an area that previously lacked research.

Who is leading the TK2d research presentations at UMDF 2025?

The disease course studies are led by M Hirano et al., while the caregiver burden study is led by A Karaa et al.

What is UCB's (UCBJY) focus regarding TK2d research?

UCB is focused on advancing the understanding of TK2d through research on disease progression, outcomes over time, and real-world insights into caregiver challenges and experiences.
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