Chair and Founder PCDResearch.org, Rare disease advocate, Drug Development Clinician, former paediatric oncology doctor. PhD - functional dependencies of osteosarcoma.
Today is #worldbronchiectasisday a condition that is still poorly understood with no approved therapies. Recent data from EMBARC, the largest series of people with bronchiectais worldwide, highlighted that airway clearance is prescribed primarily on the basis of geographical location, not evidence. This is a huge burden to put on families. Insmed Incorporated’s brenscotanib maybe closer to approval but there is still much to understand about the respiratory microbiome and the role of potential modulators. Cystic fibrosis and primary ciliary dyskinesia are both neutrophilic endotypes of bronchiectasis. While CF has modulators that address 90% of the population, PCD has worse outcomes in children, an inequitable standard of care and no approved therapies. Both populations could be combined to better understand antimicrobilal resistance. #bronchiectasis #bronchiectasisneedsmore #primaryciliarydyskinesia
Bronchiectasis is a lung condition more common in the UK than Parkinson’s or MS, yet instead of being a household name, we know very little about how it develops and progresses. Incredibly, the world’s first dedicated treatment (brensocatib) for bronchiectasis has just shown positive results in its Phase 3 clinical trial – meaning that this new drug reduced flare-ups and slowed the progression of the condition in people taking part. This research led by Professor James Chalmers and the team is an amazing leap forward for people with lung conditions, but we can’t stop there. We need to build on the momentum of this incredible discovery. That’s why this #WorldBronchiectasisDay we’re raising awareness with LifeArc of the impact of this lung condition. By working together, we can understand how best to invest in research and make the most of the real opportunity to change the way this condition is treated and managed.
