bims-tyki2d Biomed News
on Thymidine kinase 2 deficiency
Issue of 2026–04–05
three papers selected by
Zoya Panahloo, UCB



  1. CPT Pharmacometrics Syst Pharmacol. 2026 Apr;15(4): e70234
      Rare diseases affect over 300 million people globally, with approximately 75% manifesting in childhood. Their diagnosis is often delayed and approved treatments are lacking for most of the conditions. Pediatric rare diseases research is further complicated by ethical constraints and developmental diversity across childhood. Digital Twins, virtual representations of patients built from mechanistic and AI/ML models, offer a promising solution by enabling hypothesis testing, precision diagnostics, personalized therapies, and in silico trials for pediatric rare diseases. This article discusses the potential of DT applications in advancing precision medicine for pediatric rare diseases, alongside associated regulatory perspectives, modeling strategies, uncertainty analysis, as well as data, ethical and legal challenges.
    DOI:  https://doi.org/10.1002/psp4.70234
  2. Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2026 Apr 10. 43(4): 241-247
       OBJECTIVE: To investigate the current status of clinical genetics specialization development and the diagnostic and therapeutic capabilities for hereditary diseases across medical institutions in Shanghai, and to assess the necessity and feasibility of establishing training bases for clinical genetics specialists.
    METHODS: By employing a cross-sectional survey design, the Clinical Genetics Committee of Shanghai Medical Association has conducted questionnaire surveys from March to April 2025 across 54 healthcare institutions in Shanghai (including 33 tertiary hospitals and 21 secondary hospitals). The survey involved administrative departments and medical personnel from 15 clinical specialties. The survey has covered current genetic disease diagnosis and treatment practices, relevant and specialised disease types, genetic department establishment, testing capabilities, personnel teams, and training requirements.
    RESULTS: The results revealed that 78.0% of clinical departments surveyed had treated patients with hereditary disorders. Shanghai possesses diagnostic and therapeutic expertise for over 95% of hereditary diseases listed in its rare disease catalogue, reflecting both the practical clinical demand for such conditions and the city's overall diagnostic and therapeutic strengths in this field. Nevertheless, significant disparities exist in the development of genetics departments across different tiers of healthcare institutions. Resources for genetic testing capabilities (including molecular, cellular, and biochemical testing) are also unevenly distributed across different tiers of hospitals. The survey further revealed that only 26.0% of departments believe that their current physician structure fully meets the diagnostic and treatment demands. Over 90% of departments consider standard training for clinical genetic specialists necessary, with 74.0% expressing willingness to participate in establishing training bases. Based on above findings and thorough deliberation, the Clinical Genetics Committee of the Shanghai Medical Association proposes advancing specialist training and discipline development through establishing a standard training system. The committee has drafted a three-year training protocol featuring a "joint training"-centered model, recommending a pilot-first, dynamically optimized strategy for steadily advancing training base development.
    CONCLUSION: Shanghai faces substantial demand for genetic disease diagnosis and treatment, yet exhibits shortcomings in clinical genetics specialization development, resource allocation, and talent pipeline cultivation. To establish a standard training system holds significant practical importance and is underpinned by a broad demand.
    DOI:  https://doi.org/10.3760/cma.j.cn511374-20251208-00708
  3. Sociol Res Online. 2025 Mar;30(1): 278-285
      Journeys through Genomics is a series of illustrations co-produced with patients and families to communicate their experiences of seeking genomic explanations for a health condition and the wider impact on their lives. The resources are embedded within qualitative longitudinal research exploring patient's experiences of genomic medicine. This research takes place as genomic medicine becomes an integral part of mainstream care within the UK healthcare system. The depiction of genomic medicine often focuses on its technological components and the speed by which genetic code can be analysed, but here, we present a dynamic and situated understanding of the challenges genomic testing presents for patients and families. We describe the process of working with research participants and an artist to co-produce visual resources that illustrate the complexity of participant's journeys, situating genomic testing within the broader context of their lives. These resources are designed to help future patients, families, and healthcare professionals understand the process, opportunities, and challenges they may face.
    Keywords:  co-production; genomics; illustration; patient experience
    DOI:  https://doi.org/10.1177/13607804241252528