Cystic fibrosis transmembrane conductance regulator modulator administration to a F508del heterozygous infant with meconium pseudocyst and short bowel syndrome: A case report

被引：0

作者：

Stoffella, Sylvia ^{[1
]}

Ly, Ngoc ^{[2
]}

Mcgarry, Meghan ^{[2
]}

Neemuchwala, Fatima ^{[2
]}

Church, Gwynne ^{[2
]}

Chan, Marilynn ^{[2
]}

Cuneo, Addison ^{[2
]}

Omori, Makiko ^{[2
]}

Gibb, Elizabeth ^{[2
]}

机构：

[1] Univ Calif San Francisco, Dept Pharm, San Francisco, CA 94143 USA

[2] Univ Calif San Francisco, Dept Pediat, San Francisco, CA USA

来源：

PEDIATRIC PULMONOLOGY | 2024年 / 59卷 / 03期

关键词：

CFTR modulators: infant; Cystic Fibrosis; elexacaftor; ivacaftor; meconium pseudocyst; short bowel syndrome; tezacaftor;

D O I：

10.1002/ppul.26809

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

引用

页码：791 / 793

页数：3

共 4 条

[1] Normal pancreatic function and false-negative CF newborn screen in a child born to a mother taking CFTR modulator therapy during pregnancy
Fortner, Christopher N.
Seguin, Julie M.
Kay, Denise M.
[J]. JOURNAL OF CYSTIC FIBROSIS, 2021, 20 (05) : 835 - 836
[2] Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial
Heijerman, Harry G. M.
McKone, Edward F.
Downey, Damian G.
Van Braeckel, Eva
Rowe, Steven M.
Tullis, Elizabeth
Mall, Marcus A.
Welter, John J.
Ramsey, Bonnie W.
Mckee, Charlotte M.
Marigowda, Gautham
Moskowitz, Samuel M.
Waltz, David
Sosnay, Patrick R.
Simard, Christopher
Ahluwalia, Neil
Xuan, Fengjuan
Zhang, Yaohua
Taylor-Cousar, Jennifer L.
Mccoy, Karen S.
[J]. LANCET, 2019, 394 (10212) : 1940 - 1948
[3] Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
Middleton, P. G.
Mall, M. A.
Drevinek, P.
Lands, L. C.
McKone, E. F.
Polineni, D.
Ramsey, B. W.
Taylor-Cousar, J. L.
Tullis, E.
Vermeulen, F.
Marigowda, G.
Mckee, C. M.
Moskowitz, S. M.
Nair, N.
Savage, J.
Simard, C.
Tian, S.
Waltz, D.
Xuan, F.
Rowe, S. M.
Jain, R.
[J]. NEW ENGLAND JOURNAL OF MEDICINE, 2019, 381 (19) : 1809 - 1819
[4] A case report of CFTR modulator administration via carrier mother to treat meconium ileus in a F508del homozygous fetus
Szentpetery, Sylvia
Foil, Kimberly
Hendrix, Sara
Gray, Sue
Mingora, Christina
Head, Barbara
Johnson, Donna
Flume, Patrick A.
[J]. JOURNAL OF CYSTIC FIBROSIS, 2022, 21 (04) : 721 - 724

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