Access to medicines for rare diseases: beating the drum for primary ciliary dyskinesia [editorial].

Crowley, SuzanneSuzanneCrowleyAzevedo, InêsInêsAzevedoBoon, MiekeMiekeBoonBush, AndrewAndrewBushEber, ErnstErnstEberHaarman, EricEricHaarmanKaradag, BulentBulentKaradagKötz, KarstenKarstenKötzLeigh, MargaretMargaretLeighMoreno-Galdó, AntonioAntonioMoreno-GaldóMussaffi, HudaHudaMussaffiNielsen, Kim GKim GNielsenOmran, HeymutHeymutOmranPapon, Jean-FrançoisJean-FrançoisPaponPohunek, PetrPetrPohunekPriftis, KostasKostasPriftisRindlisbacher, BernhardBernhardRindlisbacherSantamaria, FrancescaFrancescaSantamariaValiulis, ArunasArunasValiulisWitt, MichalMichalWittYiallouros, PanayiotisPanayiotisYiallourosZivkovic, ZoricaZoricaZivkovicKühni, ClaudiaClaudiaKühniLucas, Jane SJane SLucas2024-10-052024-10-052020-07https://boris-portal.unibe.ch/handle/20.500.12422/55399Primary ciliary dyskinesia, a rare disease causing bronchiectasis, lacks a sound evidence base for treatment. @beatpcd proposes 1) forming a PCD European clinical trial network to address this situation and 2) conducting n-of-1 trials to access medication. https://bit.ly/3j5blfM.en600 - Technology::610 - Medicine & health300 - Social sciences, sociology & anthropology::360 - Social problems & social servicesAccess to medicines for rare diseases: beating the drum for primary ciliary dyskinesia [editorial].article10.7892/boris.1468603296400510.1183/23120541.00377-2020