Organoids are self‐organizing 3D structures grown from stem cells that recapitulate essential
aspects of organ structure and function. Here, we describe a method to establish long‐term…

Premature termination codon read-through drugs offer opportunities for treatment of multiple
rare genetic diseases including cystic fibrosis. We here analyzed the read-through efficacy …

… Vries and DD Zomer-van Ommen contributed to the acquisition of study data and revised
the manuscript. MJC Eijkemans contributed to the design of the study, analysis and …

Preclinical drug testing in primary human cell models that recapitulate disease can significantly
reduce animal experimentation and time-to-the-clinic. We used intestinal organoids to …

Background New functional assays using primary human intestinal adult stem cell cultures
can be valuable tools to study epithelial defects in human diseases such as cystic fibrosis. …

We hypothesized that people with cystic fibrosis (CF) who express CFTR (cystic fibrosis
transmembrane conductance regulator) gene mutations associated with residual function may …

… Zomer-van Ommen reviewed the manuscript on behalf of the NCFS. The NCFS and Health
… Administrative, technical or material support: DD Zomer-van Ommen. Supervision: CK van …

… Muilwijk and MJC Eijkemans; resources: DD Zomer-van Ommen and VAM … , DD Zomer-van
Ommen and CK van der Ent; writing (review and editing): D. Muilwijk, DD Zomer-van Ommen…

Background Dutch newborn screening (NBS) for Cystic Fibrosis (CF) introduced in 2011
showed a sensitivity of 90% and a positive predictive value (PPV) of 63%. We describe a study …

Objectives To develop and validate a personalized patient-reported outcome measure (PROM)
that enables a systematic assessment of the impact of care and treatment modalities on …