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1 . Academic Journal
Cystic fibrosis drug trial design in the era of CFTR modulators associated with substantial clinical benefit: stakeholders’ consensus view
저자
by
De Boeck, K.
;
Lee, T.
;
Amaral, M.
;
Drevinek, P.
;
Elborn, J.S.
, et al.
소스
In
Journal of Cystic Fibrosis
September 2020 19(5):688-695
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7 . Academic Journal
Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)
저자
by
Konstan, M.W.
;
VanDevanter, D.R.
;
Rowe, S.M.
;
Wilschanski, M.
;
Kerem, E.
, et al.
소스
In
Journal of Cystic Fibrosis
July 2020 19(4):595-601
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7 . Academic Journal
Recommendations for the classification of diseases as CFTR-related disorders
저자
by
Bombieri, C.
;
Claustres, M.
;
De Boeck, K.
;
Derichs, N.
;
Dodge, J.
, et al.
소스
In
Journal of Cystic Fibrosis
2011 10 Supplement 2:S86-S102
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7 . Academic Journal
290 Extending the success of elexacaftor-tezacaftor-ivacaftor to rare mutations—insights from CFTR structure and modulator binding
저자
by
Kerem, B.
;
Stanleigh, N.
;
Gur, M.
;
Kerem, E.
;
Wilschanski, M.
.
소스
In
Journal of Cystic Fibrosis
October 2023 22 Supplement 3:S146-S146
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7 . Academic Journal
126 A phase 1b/2a randomized, double-blind, placebo-controlled, multicenter study evaluating nebulized phage therapy in people with cystic fibrosis with chronic Pseudomonas aeruginosa pulmonary infection
저자
by
Rappo, U.
;
Kahan-Hanum, M.
;
Ussery, X.
;
Kario, E.
;
Nevenzal, H.
, et al.
소스
In
Journal of Cystic Fibrosis
October 2023 22 Supplement 3:S66-S67
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7 . Academic Journal
116 First-in-human clinical trial with SPL84, an antisense oligonucleotide for treatment of people with cystic fibrosis with the 3849 +10 Kb C - > T mutation
저자
by
Caraco, Y.
;
Wanounou, M.
;
Friedman, L.
;
Cohen, A.
;
Hart, G.
, et al.
소스
In
Journal of Cystic Fibrosis
October 2023 22 Supplement 3:S61-S61
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7 . Academic Journal
276 Preclinical and clinical safety profile of SPL84, an antisense oligonucleotide for treatment of people with cystic fibrosis carrying the 3849 +10 Kb C ->T mutation, supporting Phase 2 study initiation.
저자
by
Friedman, L.
;
Ozeri-Galai, E.
;
Cohen, A.
;
Hart, G.
;
Caraco, Y.
, et al.
소스
Journal of Cystic Fibrosis
. Sep2024 Supplement 2, Vol. 23, pS147-S148. 2p.
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7 . Academic Journal
Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice
저자
by
Castellani, C.
;
Cuppens, H.
;
Macek, M., Jr.
;
Cassiman, J.J.
;
Kerem, E.
, et al.
소스
In
Journal of Cystic Fibrosis
2008 7(3):179-196
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7 . Academic Journal
P095 A phase 1b/2a randomized, double-blind, placebo-controlled, multicentre study evaluating nebulized phage therapy in cystic fibrosis subjects with chronic Pseudomonas aeruginosa pulmonary infection
저자
by
Rappo, U.
;
Kahan-Hanum, M.
;
Ussery, X.
;
Kario, E.
;
Nevenzal, H.T.
, et al.
소스
In
Journal of Cystic Fibrosis
June 2023 22 Supplement 2:S93-S93
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7 . Academic Journal
P077 Clinical and functional efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis carrying the N1303K mutation
저자
by
Cohen-Cymberknoh, M.
;
Sadras, I.
;
Kerem, E.
;
Livnat, G.
;
Sarouk, I.
, et al.
소스
In
Journal of Cystic Fibrosis
June 2023 22 Supplement 2:S87-S87
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