Academic Journal
Fetal Transplacental Therapy to Mitigate Consequences of Cystic Fibrosis.
| Title: | Fetal Transplacental Therapy to Mitigate Consequences of Cystic Fibrosis. |
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| Authors: | Kam CW; Department of Pharmacy, University of North Carolina Hospitals, Chapel Hill, North Carolina., McKinzie CJ; Department of Pharmacy, University of North Carolina Hospitals, Chapel Hill, North Carolina., Goralski JL; Division of Pediatric Pulmonology, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina; Division of Pulmonary/Critical Care Medicine, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina. Electronic address: Jennifer_goralski@med.unc.edu. |
| Source: | Clinical therapeutics [Clin Ther] 2026 Jul; Vol. 48 (7), pp. 571-582. Date of Electronic Publication: 2026 Mar 13. |
| Publication Type: | Journal Article; Review |
| Language: | English |
| Journal Info: | Publisher: Excerpta Medica Country of Publication: United States NLM ID: 7706726 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1879-114X (Electronic) Linking ISSN: 01492918 NLM ISO Abbreviation: Clin Ther Subsets: MEDLINE |
| Imprint Name(s): | Publication: 1998- : Belle Mead, NJ, : Excerpta Medica Original Publication: Princeton, N. J., Excerpta Medica. |
| MeSH Terms: | Cystic Fibrosis*/therapy , Cystic Fibrosis*/drug therapy , Cystic Fibrosis*/genetics , Aminophenols*/therapeutic use , Aminophenols*/administration & dosage , Benzodioxoles*/therapeutic use , Benzodioxoles*/administration & dosage , Fetal Therapies*/methods, Cystic Fibrosis Transmembrane Conductance Regulator/genetics ; Humans ; Female ; Pregnancy ; Maternal-Fetal Exchange |
| Abstract: | Highly effective modulator therapy (HEMT) has been life-changing for many living with cystic fibrosis (CF), a genetic disease affecting all races and ethnicities. With diagnoses now occurring in utero for many patients, prenatal treatment of the pregnant person with HEMT to mitigate the effects of CF on the growing fetus is a hot topic of discussion. This review summarizes recent literature for fetal transplacental therapy with HEMT for providers caring for parent/fetal dyads and offers expert opinion regarding monitoring and future areas of research. (Copyright © 2026 Elsevier Inc. All rights reserved.) |
| Competing Interests: | Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: C. W. Kam: Grant funding (paid to institution) and honoraria from the Cystic Fibrosis Foundation C. J. McKinzie: Grant funding (paid to institution) and honoraria from the Cystic Fibrosis Foundation; honoraria from Vertex Pharmaceuticals, Inc. J. L. Goralski: Grant funding (paid to institution), honoraria and support for attending meetings from the Cystic Fibrosis Foundation; honoraria, travel support, and clinical trial research funding (paid to institution) from Vertex Pharmaceuticals, Inc; grant funding (paid to institution) and travel support from NIH. |
| Contributed Indexing: | Keywords: Cystic fibrosis; genetics; highly effective modulator therapy; meconium ileus; prenatal treatment; transplacental therapy |
| Substance Nomenclature: | 126880-72-6 (Cystic Fibrosis Transmembrane Conductance Regulator) 0 (Aminophenols) 0 (Benzodioxoles) |
| Entry Date(s): | Date Created: 20260314 Date Completed: 20260612 Latest Revision: 20260612 |
| Update Code: | 20260613 |
| DOI: | 10.1016/j.clinthera.2026.02.001 |
| PMID: | 41832105 |
| Database: | MEDLINE |
| ISSN: | 1879-114X |
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| DOI: | 10.1016/j.clinthera.2026.02.001 |