Results 121 to 130 of about 12,467 (245)

ePS03.2 Real-world outcomes in patients (pts) with cystic fibrosis (CF) treated with ivacaftor (IVA): analysis of 2014 US and UK CF registries

, 2016
Yongshen Bai, M. Higgins, N. Volkova, L. Bengtsson, Shuangying Tian, A. Sewall, S. Nyangoma, A. Elbert, D. Bilton   +8 more
openalex   +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

WS18.1 Combination FDL169/FDL176 is superior to tezacaftor/ivacaftor

, 2017
B. Cole, Praghnesh Bhatt, Immanuel Kwok, V. Bailey, W. An, C. Bresilla, J. Chin, Mauri E. Krouse   +7 more
openalex   +1 more source

Structural changes in lung morphology detected by MRI after modulating therapy with elexacaftor/tezacaftor/ivacaftor in adolescent and adult patients with cystic fibrosis [PDF]

, 2023
Valentina Fainardi, Kaltra Skenderaj, Andrea Ciuni, Gianluca Milanese, Michela Deolmi, Francesco Longo, Cinzia Spaggiari, Nicola Sverzellati, Susanna Esposito, Giovanna Pisi   +9 more
openalex   +1 more source

Successful Pregnancy in a Cystic Fibrosis Patient with a Severe Impairment of Lung Function Receiving Elexacaftor-Tezacaftor-Ivacaftor

, 2022
Zisis Balmpouzis, Annabelle Faure van Rossum, David Baud, Alice Panchaud, Georgia Mitropoulou, Jesica Mazza Stalder, Angela Koutsokera   +6 more
openalex   +1 more source

Hyperbilirubinemia and Gilbert's syndrome in Cystic Fibrosis patients treated with elexacaftor/tezacaftor/ivacaftor [PDF]

, 2023
Vito Terlizzi, Silviana Timpano, M. Salvi, Antonella Tosco, Alice Castaldo, Cristina Fevola, G Leonetti, P. Vitullo, Angela Sepe, Raffaele Badolato, Donatello Salvatore   +10 more
openalex   +1 more source

CARATTERIZZAZIONE DELLA FUNZIONE DI PI3K-GAMMA COME NUOVO BERSAGLIO TERAPEUTICO NELLA FIBROSI CISTICA [PDF]

, 2017
La fibrosi cistica (FC) è una malattia genetica a trasmissione autosomica recessiva (AR) causata dalla mutazione del gene CFTR (regolatore della conduttanza transmembrana della fibrosi cistica) che codifica per un canale del Cloro AMP ciclico dipendente.
Pirozzi, Flora
core  

Lumacaftor-ivacaftor in the treatment of cystic fibrosis: design, development and place in therapy

Drug Design, Development and Therapy, 2019
GJ ConnettNational Institute for Health Research, Southampton Respiratory Biomedical Research Centre, University Hospital Southampton NHS Foundation Trust, Southampton, SO16 6YD, UKAbstract: Lumacaftor-ivacaftor is a combination of two small molecule ...
Connett GJ
doaj  

Case Series: Hyperbilirubinemia under elexacaftor/tezacaftor/ivacaftor in the presence of Gilbert’s syndrome [PDF]

Julia Weitzel, Matthias Welsner, Christian Taube, Manfred Ballmann, Sivagurunathan Sutharsan   +4 more
openalex   +1 more source

Impact of Elexacaftor–Tezacaftor–Ivacaftor Therapy on Body Composition, Dietary Intake, Biomarkers, and Quality of Life in People with Cystic Fibrosis: A Prospective Observational Study [PDF]

Francisco Hevilla, Nuria Porras, María Victoria Girón, María García‐Olivares, Marina Padial, Francisco José Sánchez-Torralvo, Casilda Olveira, Gabriel Olveira   +7 more
openalex   +1 more source