New chances for pharmacotherapy of primary ciliary dyskinesia patients

(International Institute of Molecular and Cell Biology) Primary ciliary dyskinesia is a rare, genetic respiratory tract disorder leading to progressive pulmonary dysfunction. It exhibits many problems typical for orphan diseases: delayed diagnosis, limited evidence-based knowledge etc. The European Union is funding BESTCILIA (Better Experimental Screening and Treatment for Primary Ciliary Dyskinesia) to introduce uniform guidelines for PCD treatment and diagnosis across Europe. The latest notabl...