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A phase 3 open-label study of PTC923 (Sepiapterin) in Phenylketonuria

This study has been transitioned to CTIS with ID 2023-509229-31-00 check the CTIS register for the current data. Primary:• To evaluate the long-term safety of PTC923 in subjects with phenylketonuria (PKU)• To evaluate changes from baseline in…

Phase 2 Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sotatercept (MK-7962) in Children from 1 to Less Than 18 Years of Age With PAH on Standard of Care

This study has been transitioned to CTIS with ID 2023-504861-22-00 check the CTIS register for the current data. This study will assess the safety and tolerability of sotatercept in pediatric participants with PAH WHO Group 1 who receive PAH…

Prospective Analysis of Pharmacokinetic Infliximab data in Paediatric Inflammatory Bowel Disease patients

This study has been transitioned to CTIS with ID 2023-507352-72-00 check the CTIS register for the current data. The primary study objective of our study is to assess the efficacy of an IFX intensified induction scheme vs. a standard dosing schedule…

A 5-year Natural History Study in LAMA2-related muscular dystrophy and SELENON-related myopathy: the extended LAST STRONG Study

With the extended LAST STRONG Study, we aim to (1) collect 3-year and 5-year natural history data, (2) implement the natural history data collection into clinical care and international guidelines (reach trial readiness), and (3) do in-depth…

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

This study has been transitioned to CTIS with ID 2024-515025-28-00 check the CTIS register for the current data. To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in…

'Study on infant Adiposity development To Understand the Role of early life Nutrition - the Sophia Saturn Study'

Primary Objective:To investigate change in fat mass index between 6 and 12 months of age in infants receiving Nuturis® formula feeding versus standard formula feeding during the first 6 months of life.Secondary Objectives:- To investigate change in…