Results 171 to 180 of about 1,231,622 (330)

CFTR Modulator Therapy For Cystic Fibrosis [PDF]

, 2020
IntroductionCystic fibrosis (CF) is the most common life-limiting autosomal recessive condition in Caucasians, affecting the respiratory system, digestive tract and all exocrine glands.
Dobrudjanska, N., Galabova, M., Georgieva, M., Koleva, K., Nikolova, M., Rasheva, N.   +5 more
core   +1 more source

The validated French CFAbd‐Score reveals a lower burden of gastrointestinal symptoms in patients on Elexacaftor/Tezacaftor/Ivacaftor

Journal of Pediatric Gastroenterology and Nutrition, Volume 82, Issue 1, Page 24-32, January 2026.
Abstract Objectives Multiorgan abdominal involvement is a hallmark of Cystic fibrosis (CF). The CFAbd‐Score© is the first CF‐specific gastrointestinal patient reported outcome‐measure (PROM) developed following FDA‐guidelines. The PROM has proved to sensitively differentiate people with CF (pwCF) from healthy controls (HC).
Isabelle Sermet‐Gaudelus, Pauline Sadrieh, Anne Sophie Bonnel, Raphael Enaud, Nathalie Wizla, Jeanne Languepin, Ambre Nangui, Audrey Deelawon, Johanna Rave, Samira Dabelow, Anton Barucha, Carlos Zagoya, Franziska Duckstein, Jochen Georg Mainz   +13 more
wiley   +1 more source

Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation

Children
Background/objectives: Cystic fibrosis (CF) is a life-limiting genetic disorder affecting multiple organ systems. This study compared clinical outcomes, hospitalization rates, and survival between children and adolescents with CF who received CFTR ...
Eman A. Toraih, Hassan A. Malik, Rahib K. Islam, Humza A. Pirzadah, Ahmed Abdelmaksoud, Rami M. Elshazli, Paul Antwi Boasiako, Shehab Ahmed Alenazi, Angelique Dabel, Jessan A. Jishu, Bandar T. Alenezi, Hani Aiash, Manal S. Fawzy   +12 more
doaj   +1 more source

302 Clinical response to modulator therapy in patients homozygous for G27V CFTR

, 2023
J. Brewington, R. O’Shaughnessy, Kelly Thornton, A. Ostmann, J. Meeker, H. V. Morgan, Laura L. Walkup, Matthew M. Willmering, Jason C. Woods, Gary L. McPhail   +9 more
openalex   +1 more source

Mastering Organoid Growth: A Complete Guide to Overcoming Methodological Challenges

MedComm, Volume 7, Issue 1, January 2026.
Organoids offer transformative 3D models that precisely mimic human organ architecture and function. This review details the latest methodology, emphasizing the cultivation of liver and other abdominal organoids. We analyze technological challenges (e.g., nutrient delivery, scalability, diversity) and provide practical insights for new researchers ...
Chunbao Jiao, Omer Faruk Karakaya, Neda Dadgar, Chase J. Wehrle, Zahra Massoud, Hanna Hong, Robert L. Fairchild, Nic Leipzig, Federico Aucejo, Wen Wee Ma, Jan Joseph Melenhorst, Sofia Ferreira Gonzales, Andrea Schlegel   +12 more
wiley   +1 more source

326 Modulator response of rare CFTR missense variants not approved for elexacaftor-tezacaftor-ivacaftor

, 2023
Deepak Joshi, András Rab, Eric J. Sorscher, Jeong S. Hong   +3 more
openalex   +1 more source

Organoids in Cancer Research and Regenerative Medicine: Current Status, Challenges, and Future Prospects

MedComm, Volume 7, Issue 1, January 2026.
The management of cancer and tissue regeneration remains a substantial challenge to global health. Organoids, which are stem cell‐derived three‐dimensional multicellular structures, are revolutionizing biomedical research. Therefore, this review highlights their applications in cancer research and regenerative medicine, while discussing current ...
Ruiyang Li, Yuezhou Wu, Zhu'anzhen Zheng, Fengjin Zhou, Ke Xu, Jiacan Su   +5 more
wiley   +1 more source

Chronic rhinosinusitis in the era of CFTR modulator therapy.

Journal of Cystic Fibrosis, 2023
Roy Gavin Stone, C. Short, Jane C. Davies, Paul McNally   +3 more
semanticscholar   +1 more source

Expanding Cystic Fibrosis Registries to the Rest of the World

Pediatric Pulmonology, Volume 61, Issue 1, January 2026.
ABSTRACT Challenge Cystic fibrosis (CF) is a global challenge. The epidemiological knowledge is incomplete and focused on patient registries in the United States, Canada, Europe, Australia and New Zealand, Brazil, and South Africa. To complete the global picture of CF, we have to learn from each individual with CF, as well as each cohort and population.
Lutz Naehrlich
wiley   +1 more source

Epithelial responses to CFTR modulators are improved by inflammatory cytokines and impaired by antiinflammatory drugs

JCI Insight
Cystic fibrosis (CF) is a genetic disorder that disrupts CF transmembrane conductance regulator (CFTR) anion channels and impairs airway host defenses. Airway inflammation is ubiquitous in CF, and suppressing it has generally been considered to improve ...
Tayyab Rehman, Alejandro A. Pezzulo, Andrew L. Thurman, Rachel L. Zemans, Michael J. Welsh   +4 more
doaj   +1 more source