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

Author:

Szentpetery SylviaORCID,Foil KimberlyORCID,Hendrix SaraORCID,Gray Sue,Mingora ChristinaORCID,Head Barbara,Johnson Donna,Flume Patrick A.

Publisher

Elsevier BV

Subject

Pulmonary and Respiratory Medicine,Pediatrics, Perinatology and Child Health

Reference17 articles.

1. Annual Data Report [Internet]. Cystic Fibrosis Foundation. 2020.

2. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del);Southern;Cochrane Database Syst Rev,2020

3. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2–5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study;Davies;Lancet Respir Med,2016

4. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study;Rosenfeld;Lancet Respir Med,2018

5. Normal pancreatic function and false-negative CF newborn screen in a child born to a mother taking CFTR modulator therapy during pregnancy;Fortner;J Cyst Fibros,2021

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