WG1 Basic science (Lead: Dominic Norris) unites basic researchers to identify the infrastructure and data required to develop novel treatments for PCD. Projects include: 

  • Identifying and validating existing models of PCD
  • Identifying new models to test genotype specific therapies
  • Building the infrastructure with which cells, tissues & tools for studies in vitro and in vivo can be exchanged
  • Collaborating on preclinical treatment studies,
  • Advancing the understanding of pathogens in PCD
  • Standardizing and improving diagnostic tests

WG2 Epidemiology (Lead: Claudia Kuehni). Projects include:

  • Increasing the number of patients included in the iPCD Cohort and the European PCD registry
  • Actively recruiting adult physicians and study relevant topics  (severe lung disease, ENT, fertility treatments)
  • Developing standardized forms for prospective multicentre data collection
  • Performing data meta-analyses to describe changes in PCD phenotype, severity over life course, patterns of disease progression etc.

WG3 Clinical care (Lead: Kim Nielsen). Projects include:

  • An international survey of paediatric and adult physicians to assess current practices including respiratory, ENT and fertility
  • Identifying areas of clinical practice associated with good outcome  (with WG2)
  • Developing a consensus statement for infection control
  • Identifying priority areas for research on therapeutic drugs  (e.g. mucolytic agents, decongestants, mucociliary clearance)

WG4: Clinical trials (Lead: Philipp Latzin). Projects include: 

  • Identifying relevant outcome measures for clinical trials in children and adults  (physiological/lung function measures that are sensitive and responsive to disease progression and therapeutic interventions) 
  • Developing a consensus statement for defining respiratory exacerbations as an outcome measure for clinical trials
  • Draw on the expertise gained from the randomised clinical trial in PCD in BESTCILIA
  • An investigation to validate lung function spirometry as an outcome measure in PCD