Results 51 to 60 of about 4,770 (180)

Nanomolar-potency 'co-potentiator' therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants. [PDF]

, 2019
Available CFTR modulators provide no therapeutic benefit for cystic fibrosis (CF) caused by many loss-of-function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel, including N1303K. We previously introduced the
Finkbeiner, Walter E, Haggie, Peter M, Nielson, Dennis W, Phuan, Puay-Wah, Rivera, Amber A, Tan, Joseph-Anthony, Verkman, Alan S, Zlock, Lorna   +7 more
core  

Proliferative activity of antigen-specific CD154+ T cells against bacterial and fungal respiratory pathogens in cystic fibrosis decreases after initiation of highly effective CFTR modulator therapy [PDF]

, 2023
Background: Together with impaired mucociliary clearance, lung disease in cystic fibrosis (CF) is driven by dysregulation of innate and adaptive immunity caused by dysfunctional CFTR (Cystic Fibrosis Transmembrane Conductance Regulator), leading to ...
Bacher, Petra, Eschenhagen, Patience N, Grehn, Claudia, Mainz, Jochen G, Scheffold, Georg Alexander, Schwarz, Carsten   +5 more
core   +1 more source

Determining the Minimal Clinically Important Difference of the 40‐Item Smell Identification Test in People With Cystic Fibrosis

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) are highly prevalent among people with cystic fibrosis (PwCF) and negatively impact quality of life. The 40‐item Smell Identification Test (SIT) is widely used to assess psychophysical olfaction, but a CF‐specific minimal clinically important difference (MCID) has not been ...
Eugene Oh, Jessa E. Miller, Michelle J. Lee, Anna Zemke, David Baraghoshi, Matthew J. Strand, Jeremiah A. Alt, Todd E. Bodner, Michael Chang, Naweed I. Chowdhury, Patricia H. Eshaghian, Anne E. Getz, Jennifer L. Goralski, David A. Gudis, Peter H. Hwang, Ashoke Khanwalkar, Adam J. Kimple, Jivianne T. Lee, Douglas A. Li, Jess C. Mace, Jayakar V. Nayak, Jonathan B. Overdevest, Zara Patel, Daniella K. Safatian, Rodney J. Schlosser, Brent Senior, Amanda L. Stapleton, Timothy L. Smith, Zachary M. Soler, Jeffrey D. Suh, Grant A. Turner, Marilene B. Wang, Milene T. Saavedra, Jennifer L. Taylor‐Cousar, Daniel M. Beswick   +34 more
wiley   +1 more source

Impact of elexacaftor-tezacaftor-ivacaftor in lung transplantation for cystic fibrosis in the United States

JHLT Open
Background: Cystic fibrosis (CF) is an autosomal recessive condition leading to progressive lung disease and often necessitating lung transplantation. Historically, CF has been one of the leading indications for lung transplants in the United States. The
Tahuanty A. Pena, MD, MS, Brittany Wright, PharmD, Kalpaj R. Parekh, MBBS, Julia Kleney-Tait, MD, PhD   +3 more
doaj   +1 more source

Bioactive Thymosin Alpha-1 Does Not Influence F508del-CFTR Maturation and Activity. [PDF]

, 2019
Deletion of phenylalanine 508 (F508del) in the cystic fibrosis transmembrane conductance regulator (CFTR) anion channel is the most frequent mutation causing cystic fibrosis (CF). F508del-CFTR is misfolded and prematurely degraded. Recently thymosin a-1 (
Armirotti, Andrea, Braccia, Clarissa, Cholon, Deborah M, Galietta, Luis JV, Gentzsch, Martina, Guidone, Daniela, Hanrahan, John W, Lukacs, Gergely L, Matthes, Elizabeth, Pedemonte, Nicoletta, Phuan, Puay-Wah, Tomati, Valeria, Veit, Guido, Verkman, Alan S   +13 more
core  

Long‐term impact of Elexacaftor/Tezacaftor/ivacaftor on pulmonary, nutritional and metabolic outcomes in homozygous F508del cystic fibrosis patients: A real‐world cohort study

British Journal of Clinical Pharmacology, EarlyView.
Aim Elexacaftor/tezacaftor/ivacaftor (ETI) has markedly improved cystic fibrosis (CF) outcomes. However, its long‐term impact on nutrition, metabolism and liver health remains underexplored. We assessed 30‐month changes in pulmonary, nutritional, metabolic and inflammatory markers in people with CF (PwCF) homozygous for F508del.
Nicola Perrotta, Luigi Angelo Fiorito, Roberta Vescovo, Anna Virgilio, Giulia Amato, Giuseppe Cimino   +5 more
wiley   +1 more source

Cystic fibrosis-related diabetes in the era of modern treatment using CFTR modulators in pediatric patients—a systematic review

Frontiers in Pediatrics
BackgroundCystic fibrosis-related diabetes (CFRD) is a common comorbidity in cystic fibrosis (CF), particularly in the pediatric population. As cystic fibrosis transmembrane conductance regulator modulators (CFTRm) become widely used, there is growing ...
Anna Pietrzykowska, Jerzy Darmoń, Katarzyna Szymocha, Wiktoria Donocik, Mateusz Tarasiewicz, Julia Stadnik, Przemysława Jarosz-Chobot   +6 more
doaj   +1 more source

Farmacogenética en Fibrosis Quística. [PDF]

, 2017
La fibrosis quística (a partir de ahora referida como FQ) fue descrita por primera vez en 1936 como una entidad separada. También recibía el nombre de “mucoviscidosis” debido a la acumulación de espesas secreciones mucosas que bloqueaban las vías aéreas,
Oraá Pérez, Javier
core  

Comparison of Single‐Breath and Multi‐Breath Xe‐MRI in the Longitudinal Assessment of Treatment in Children With Cystic Fibrosis

Journal of Magnetic Resonance Imaging, EarlyView.
ABSTRACT Background Elexacaftor/tezacaftor/ivacaftor (ETI) is a current standard therapy for pediatric cystic fibrosis (CF). Multiple‐breath washout 129Xe MRI (MBW Xe‐MRI) is improved following 1 month of treatment. However, the utility of MBW Xe‐MRI over extended ETI treatment and its comparison to single‐breath Xe‐MRI and pulmonary function tests ...
Faiyza S. Alam, Samal Munidasa, Brandon Zanette, Daniel Li, Sharon Braganza, Felix Ratjen, Giles Santyr   +6 more
wiley   +1 more source

Novel reaction to new cystic fibrosis medication Trikafta

Clinical Case Reports, 2021
We present a novel case of an urticaria multiforme‐type drug reaction to the new cystic fibrosis medication Trikafta (elexacaftor + tezacaftor + ivacaftor).
Julian Stashower, Patrick Carr, Virginia Miller, Barrett Zlotoff   +3 more
doaj   +1 more source