Primary ciliary dyskinesia (PCD) is a rare inherited disorder of ciliary function causing chronic airway infection and bronchiectasis, and its management has largely been extrapolated from cystic fibrosis and bronchiectasis care. The most important trial-based advance is azithromycin: the first international randomized controlled trial in PCD showed it reduces exacerbations, supporting long-term macrolide maintenance. Supportive care — airway clearance, mucolytics such as hypertonic saline, and antibiotics for infection — remains central. Looking ahead, gene therapy to restore ciliary function is a promising but early strategy, and trials such as Move-PCD are testing whether individualized physical-activity programs improve quality of life and lung function. PCD still lacks a disease-modifying, mechanism-targeted therapy analogous to the CFTR modulators in CF, which remains the central unmet need.
Further reading: azithromycin in PCD (BESTCILIA randomized trial); reviews of PCD management and emerging gene therapy; Move-PCD activity trial.