bims-tyki2d Biomed News
on Thymidine kinase 2 deficiency
Issue of 2026–07–19
four papers selected by
Zoya Panahloo, UCB



  1. Public Health Genomics. 2026 Jul 14. 1-21
       BACKGROUND: The diagnostic journey for rare diseases (RDs) often involves lengthy delays and significant burdens on patients and their family. Genetic newborn screening (NBS) for RDs offers a potential opportunity for early diagnosis and treatment.
    AIMS: This study explores factors influencing parental decisions regarding genetic NBS for RDs participation in two European countries (Italy and Germany).
    METHODS: This mixed methods qualitative study is composed of focus group discussions and ranking exercises. It was conducted with parents seeking genetic testing (n=5) and expecting parents from the general population (n=11). All participants were recruited in spring 2024 in Germany, and winter 2024 in Italy via clinical centers contact from genetic centers and obstetric services. Thematic analysis was performed by two independent coders to identify main themes and sub-themes.
    RESULTS: Three key themes were constructed: 1) the complexity of the decision-making process, 2) the importance of certainty and disease characteristics, and 3) the anticipation of negative consequences. Participants trusted healthcare professionals to guide their decisions, meanwhile emphasized the need for extensive education and comprehensive support systems. Concerns about test accuracy, potential psychological impact of uncertain or positive results, and anticipated social implications were also reported.
    CONCLUSION: These findings highlight the nuanced considerations surrounding genetic NBS for RDs implementation and the necessity of addressing parental experiences in clinical practice and policy development. Participants emphasized the importance of accessible education and clear communication, support of trusted healthcare professional, and ethical reflection are essential for the equitable integration of genetic NBS into newborn care.
    DOI:  https://doi.org/10.1159/000553234
  2. J Med Internet Res. 2026 Jul 16. 28 e106582
       Unlabelled: Rare diseases are often critically underfunded, leaving many patients without timely diagnosis and treatment. In this News and Perspectives article, JMIR Correspondent Simon Spichak, who was a 2025 recipient of the National Press Foundation Rare Disease Reporting Fellowship, reports on advances in AI modeling that may offer new promise for rare disease detection and care.
    Keywords:  artificial intelligence; diagnosis, computer-assisted; foundation models; frontier models; genetic variation; machine learning; rare diseases
    DOI:  https://doi.org/10.2196/106582
  3. J Genet Couns. 2026 Aug;35(4): e70258
      Rare diseases (RDs) are often subject to diagnostic delays due to their low prevalence, clinical variability, and limited professional awareness. This scoping review aimed to map the literature on these delays, examining their clinical, emotional, and socioeconomic consequences. Conducted in accordance with the PRISMA-ScR guidelines, the review identified 23 studies published between 2010 and 2025. The included studies spanned 13 countries, with a notable concentration in Europe and increasing publication trends in recent years, reflecting growing international recognition of the challenges associated with delayed diagnosis. Across diverse study designs and disease contexts, commonly reported consequences included misdiagnosis and inappropriate treatment, psychological distress such as anxiety and frustration, disease progression, increased healthcare utilization, social isolation, reduced quality of life, and financial burden. These findings underscore the broad clinical and psychosocial impact experienced by patients during delayed diagnostic processes. Reducing diagnostic delay in RDs requires coordinated public health efforts, improved diagnostic infrastructure, and greater investment in professional training. Such efforts are essential to ensure earlier diagnosis, improve health outcomes and quality of life, as well as to enable timely access to genetic counseling to better support patients and families.
    Keywords:  delayed diagnosis; neglected diseases; rare diseases
    DOI:  https://doi.org/10.1002/jgc4.70258