How exciting! ReCode Therapeutics have developed an mRNA treatment that hopes to restore ciliary function in patients with pathogenic mutations in the DNAI1 gene We have everything crossed that the Phase 1 trial goes well! Read more 👇👇 recodetx.com/recode-therape…

#DidYouKnow BBC News (UK) has a dedicated #RareDisease page! Read rare disease stories from across the nation in one, centralised place bbc.co.uk/news/topics/cz…

It has been a huge joint effort to get to this point: the first fully decentralised non pharmacological clinical trial in #PCD. Thanks to the amazing team Aparito (particularly Elisa Ferrer Mallol & Dr Elin Haf Davies) for believing in us. If you have PCD check it out! 👇#research

Big day today - still raising awareness of PCD PCD Support UK Ciliopathy Alliance #Coronation #raredisease

It's been a rough few months, with nearly 4 weeks of hospital admissions since end of Jan. Here's me getting my last dose last night before heading home! I can't thank the amazing staff at Royal Brompton and Harefield enough. Also loving my new accessible Porto&bello hoodie! #PCD #SpecialistCare

Me on my way to our 2023 #PCD medical board meeting (on 5 hours sleep)...

At the PCD Support UK medical board in The BMA where I display a poster about the use of AI for the diagnosis of PCD. Royal Brompton and Harefield NHLI National Institute for Health and Care Research BEAT-PCD (ERS CRC)

Medmin: My hospital discharge summary was inaccurate but the doc said he was new and didn't know how to make edits, so couldn't change it. This was sent to my GP surgery who have now taken 2 of my essential meds off my repeat, and are refusing to put them back on. Ffs 🤯 #PCD

🫁The thing with #PCD is that you can do everything right & it's still not enough. I tell this to parents and fellow PCDers all the time, & yet a phone call from hospital still has me asking what I could have done differently. Channeling sad dog vibes...

Calling all paediatric physiotherapist 🗣️ Come join our amazing data science team to develop and validate new digital biomarkers 🙌 Apply today 👇

Just one of the many ways we make your donations count 👇

People with PCD: please fill out this survey! Great to have an opportunity to shape the future of research in PCD and hope it leads to new avenues being explored (spoiler alert: it includes gastroenterology, which people often ask @pcdsupportuk about!) #pcd #participatoryresearch