by Michele Manion | Oct 24, 2019 | Advocacy
Vertex Announces FDA-Approval of New Triple Drug Compound Effective for the Most Common Mutation in Cystic Fibrosis Yesterday, the CF Foundation and Vertex announced that the FDA had approved a triple-drug compound that is designed to be effective for the most...
by Michele Manion | Oct 9, 2019 | RESEARCH
We are delighted to announce that the Genetic Disorders of Mucociliary Clearance Consortium (GDMCC) has been re-funded for another five years (years 15-20) of research on PCD and related disorders. Here is the official announcement from the National Center for...
by Michele Manion | Oct 23, 2017 | Diagnosis, Genetic Testing, Genetics
PCD diagnosis continues to be challenging. All current testing options available to aid in PCD diagnosis (biopsy with TEM, nasal nitric oxide measurement, high-speed videomicroscopy, immunofluorescent assay of ciliary proteins) have limitations and do not work in all...
by Michele Manion | Sep 22, 2017 | Advocacy
The proposed Graham-Cassidy bill is bad for individuals with rare disorders. Our colleagues at the United Mitochondrial Disease Foundation (UMDF) engage an independent legislative advisory group, Holland & Knight, to assess the impact of proposed legislation on...
by Michele Manion | Aug 28, 2017 | EDUCATION, Meetings, RESEARCH
Summary PCD on the Move 2017 (OTM 2017), the fourth annual North American scientific meeting dedicated to primary ciliary dyskinesia (PCD), focused on how to move ciliary/PCD science into practical applications to improve the quality of life for individuals with PCD....
