Five years later, I remain deeply grateful to Prof. Bruce Stanton for his generous praise of my sole-author paper. For those interested, the publication is available here: doi.org/10.3389/fpha...

#tbt #cysticfibrosis #drugdevelopment #precisionmedicine #cftrmodulators

Illustration of a smiling red liver on a light teal background with text: “Liver transplants may become less common since approval of CFTR modulators in CF.”

See how CF meds help: https://bit.ly/48d0IlR

A new study suggests that CFTR modulators may reduce or delay the need for liver transplants in people with cystic fibrosis.

#CysticFibrosisNews #CysticFibrosis #CFTRModulators #CFLiverDisease #Bionews

Nationwide Children's Pediatric #Pulmonology team is proud to be contributing to #CHEST2025! From #CFTRmodulators & #asthma phenotypes to cutting-edge imaging & #SleepMedicine innovations, our experts are sharing research, insights & clinical strategies in multiple sessions.
https://bit.ly/47kyEMQ

INTEGRATING GENOMIC AND FUNCTIONAL TESTING TO IMPROVE CFTR MODULATOR RESPONSE PREDICTION IN CHILDREN WITH CYSTIC FIBROSIS
Allan, K. M., Chew, Z. A. et al.
Paper
Details
#GenomicTesting #CFTRModulators #CysticFibrosisResearch

Divergent Neurobehavioral Effects of CFTR Modulators Elexacaftor and Ivacaftor in Mice
Du, J., Fagan, A. et al.
Paper
Details
#CFTRModulators #NeurobehavioralEffects #ElexacaftorIvacaftorResearch

Rescuing Trafficking Mutants of the ATP-binding Cassette Protein, ABCA4, with Small Molecule Correctors as a Treatment for Stargardt Eye Disease - PubMed Stargardt disease is the most common form of early onset macular degeneration. Mutations in ABCA4, a member of the ATP-binding cassette (ABC) family, are associated with Stargardt disease. Here, we ha...

#tbt #sciencefacts
In this #experimentalstudy, we demonstrated that certain #CFTRmodulators rescue misfolded variants in #ABCA4, a protein involved in macular degeneration. These may represent promising #therapeutics for #Stargard #eyedisease 🧑🏻‍🔬🧬💊
#drugdevelopment

pubmed.ncbi.nlm.nih.gov/26092729/

#cftrmodulators have been a transformation in lives of many people with #cysticfibrosis but we are not done until it's done for everyone! 🧑🏻‍🔬🧬🫁
#precisionmedicine

PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR The deletion of a phenylalanine at position 508 (p.Phe508del) in the CFTR anion channel is the most prevalent variant in people with Cystic Fibrosis (…

Our new paper has been published: "PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR" in European Journal of Pharmacology 👨‍🔬🫁🔬🧫💊
#CysticFibrosis #PersonalizedMedicine #cftrmodulators #mechanismofaction

www.sciencedirect.com/science/arti...

Organic Synthesis and Current Understanding of the Mechanisms of CFTR Modulator Drugs Ivacaftor, Tezacaftor, and Elexacaftor The monogenic rare disease Cystic Fibrosis (CF) is caused by mutations in the gene encoding the CF transmembrane conductance (CFTR) protein, an anion channel expressed at the apical plasma membrane of...

Our new paper has been published: "Organic Synthesis and Current Understanding of the Mechanisms of CFTR Modulator Drugs Ivacaftor, Tezacaftor, and Elexacaftor" in Molecules MDPI 🧑‍🔬🔬🧫🧬💊

#cysticfibrosis #personalizedmedicine #cftrmodulators #addingtomorrows

www.mdpi.com/1420-3049/29...

Laboratory Tools to Predict CFTR Modulator Therapy Effectiveness and to Monitor Disease Severity in ... The implementation of cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has been attaining remarkable therapeutic outcomes for CF, a life-threateni...

Our new paper has been published: "Laboratory Tools to Predict CFTR Modulator Therapy Effectiveness and to Monitor Disease Severity in Cystic Fibrosis" in @JPM_MDPI 🧑‍🔬🔬🧫🧬💊 #cysticfibrosis #personalizedmedicine #cftrmodulators #invitromodels #addingtomorrows

www.mdpi.com/2075-4426/14...