SNSF Ambizione Grant on upper airways disease in Primary Ciliary Dyskinesia

SNSF Ambizione Grant

22.08.2019 – Myrofora Goutaki  has been awarded a Swiss National Science Foundation Ambizione grant to characterise and provide first evidence for the management of upper airways disease in patients with primary ciliary dyskinesia in a large multi-national project. Primary ciliary Dyskinesia (PCD) is a rare genetic, multi-organ disease, which affects primarily the airways.  Almost all PCD patients suffer from upper respiratory symptoms (ear-nose-throat, ENT) that are often severe, yet research related to the upper airways is scarce.

Myrofora will characterise ENT disease in PCD patients of all ages, study how it is associated with lower respiratory disease, and identify determinants of disease prognosis. To achieve this, she will develop and analyse data from EPIC-PCD (ENT Prospective International Cohort of PCD patients), a prospective multi-centre cohort embedded in routine clinical care. In addition, in collaboration with PCD experts in several European centres and with the Clinical Trials Unit in Bern, she will lead a multi-centre clinical trial to provide first evidence for the management of chronic rhinosinusitis in PCD patients, one of the most common problems of these patients that affects severely their quality of daily life. During the project, she will also collaborate closely with the PCD patient organisations in Europe.

This project aligns with national and European efforts to intensify collaborative research in rare diseases, focusing on an important area of PCD where more efforts are needed. It combines observational and interventional approaches to understand ENT disease in PCD and provide new, robust, clinically relevant evidence to better inform medical management of patients.