Abstract
Primary ciliary dyskinesia (PCD) is a rare heterogenous condition that causes progressive suppurative lung disease, chronic rhinosinusitis, chronic otitis media, infertility and abnormal situs. ‘Better Experimental Approaches to Treat Primary Ciliary Dyskinesia’ (BEAT-PCD) is a network of scientists and clinicians coordinating research from basic science through to clinical care with the intention of developing treatments and diagnostics that lead to improved long-term outcomes for patients. BEAT-PCD activities are supported by EU funded COST Action (BM1407). The second BEAT-PCD conference, and third PCD training school were held jointly in April 2017 in Valencia, Spain. Presentations and workshops focussed on advancing the knowledge and skills relating to PCD in: basic science, epidemiology, diagnostic testing, clinical management and clinical trials. The multidisciplinary conference provided an interactive platform for exchanging ideas through a program of lectures, poster presentations, breakout sessions and workshops. Three working groups met to plan consensus statements. Progress with BEAT-PCD projects was shared and new collaborations were fostered. In this report, we summarize the meeting, highlighting developments made during the meeting.
Highlights
Primary ciliary dyskinesia (PCD) is a rare syndrome characterized by impaired mucociliary clearance due to abnormal ciliary function
For example with BESTCILIA, an international PCD cohort of over > 3000 patients providing infrastructure for epidemiological studies [7] and a PCD registry to support future clinical trials have been established [8], the first multinational clinical trial of a treatment for PCD is analysing results [9], and we have developed, validated and translated quality of life tools (QOL-PCD) for use as outcome measures in trials [10,11,12]
Since some of the members of the BEAT-PCD group have previous experience in gene therapy (GT) and others in the development of experimental models of the disease, we proposed the possibility of using these models to evaluate the efficiency of the GT techniques in the treatment of the disease
Summary
From 2nd BEAT-PCD Conference and 3rd PCD Training School Valencia, Spain. 18-21 April 2017
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