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A phase 3 extension study of Ataluren (PTC124) in patients with Nonsense Mutation Cystic Fibrosis

Primary objectiveTo evaluate the long-term safety of 10-, 10-, 20-mg/kg ataluren in patients with nonsense mutation cystic fibrosis (nmCF), who previously participated in pivotal study PTC124-GD-021-CF, as determined by adverse events and laboratory…

Newborns with Congenital Diaphragmatic hernia: inhaled Nitric Oxide versus intravenous Sildenafil: ;an international randomized controlled trial

To determine the most effective drug (iNO or IV sildenafil) for the treatment of PH in newborns with CDH and to reach evidence based dosing of this drug. Subsequently this drug will be added to the international guidelines for the treatment of…

A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease

Primary objectiveThe primary objective of the study is to determine the long term safety and tolerability of lucerastat in subjects with Fabry disease (FD).Secondary objectives* To evaluate the effect of lucerastat on renal function and cardiac…

An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema

This study has been transitioned to CTIS with ID 2024-510777-18-00 check the CTIS register for the current data. The primary objective of the study is to evaluate the long-term safety of SC administration of CSL312 in the prophylactic treatment of…