Efficacy and tolerance of intravenous methylprednisolone pulses in children with severe bronchopulmonary dysplasia requiring respiratory support.

Objectives: Bronchopulmonary dysplasia (BPD) is a chronic lung disease that affects premature babies and contributes to their morbidity. French BPD guidelines suggest the use of intravenous methylprednisolone pulses (IVMP) for those who require respiratory support (RS) following 36 weeks postmenstrual age (PMA). However, there is scant data on the efficacy and tolerance for this indication.

The expanded French compassionate programme for elexacaftor-tezacaftor-ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study.

Background: Elexacaftor-tezacaftor-ivacaftor has been approved in Europe for people with cystic fibrosis with at least one F508del CFTR variant. Additionally, it is approved by the US Food and Drug Administration (FDA) for people with cystic fibrosis with at least one of 177 rare variants. The aims of this study were to describe the clinical response to elexacaftor-tezacaftor-ivacaftor for people with cystic fibrosis without a F508del CFTR variant in France and to determine CFTR variant responsiveness to elexacaftor-tezacaftor-ivacaftor based on the observed clinical response.