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Treatment of Fetal Cystic Fibrosis With Cystic Fibrosis Transmembrane Conductance Regulator Modulation Therapy

ANNALS OF INTERNAL MEDICINE (2023)

Worldwide rates of diagnosis and effective treatment for cystic fibrosis

Jonathan Guo et al.

Summary: This study identifies gaps in access to cystic fibrosis (CF) treatment, particularly in low- and middle-income countries. The results reveal a significant burden of undiagnosed or untreated CF patients in many countries where disease-modifying drugs are unavailable.

JOURNAL OF CYSTIC FIBROSIS (2022)

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Article Respiratory System

Drug exposure to infants born to mothers taking Elexacaftor, Tezacaftor, and Ivacaftor

Bethany Collins et al.

Summary: The study showed that ETI compounds can be transferred through the placenta to cord blood of fetuses in mother-infant pairs continuing ETI therapy during pregnancy, and then further passed on to infants through breast milk postnatally.

JOURNAL OF CYSTIC FIBROSIS (2022)

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Article Respiratory System

A case report of CFTR modulator administration via carrier mother to treat meconium ileus in a F508del homozygous fetus

Sylvia Szentpetery et al.

Summary: This case report describes the treatment of a pregnant woman with F508del homozygous fetus using elexacaftor-tezacaftor-ivacaftor (ETI). The treatment resulted in resolution of meconium ileus (MI) and the delivery of a healthy baby. The mother continued ETI treatment while breastfeeding.

JOURNAL OF CYSTIC FIBROSIS (2022)

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Article Respiratory System

Worldwide rates of diagnosis and effective treatment for cystic fibrosis

Jonathan Guo et al.

Summary: This study aims to identify gaps in access to cystic fibrosis (CF) treatment globally. The findings suggest that in some countries, disease-modifying drugs are not available, leading to unmet needs among CF patients. The study highlights the potential to improve diagnosis and treatment rates for CF.

JOURNAL OF CYSTIC FIBROSIS (2022)

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Review Genetics & Heredity

Prenatal genetic testing for cystic fibrosis: a systematic review of clinical effectiveness and an ethics review

Sharon J. M. Kessels et al.

GENETICS IN MEDICINE (2020)

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Treatment of Fetal Cystic Fibrosis With Cystic Fibrosis Transmembrane Conductance Regulator Modulation Therapy

相关参考文献

Worldwide rates of diagnosis and effective treatment for cystic fibrosis

Drug exposure to infants born to mothers taking Elexacaftor, Tezacaftor, and Ivacaftor

A case report of CFTR modulator administration via carrier mother to treat meconium ileus in a F508del homozygous fetus

Worldwide rates of diagnosis and effective treatment for cystic fibrosis

Prenatal genetic testing for cystic fibrosis: a systematic review of clinical effectiveness and an ethics review

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