Ospedale Pediatrico Bambino Gesu | Dipartimento di Oncoematologia e Terapia Cellulare e Genica

The aim of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of filgotinib as a treatment for UC in children an...

This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.The...

This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The...

The AFFINITY Study is a phase 2, open-label, basket study to evaluate the efficacy and safety of atrasentan in patients with proteinuric glomerular d...

This is a Phase 1-2, multicenter, international, single-arm, open-label study designed to identify a recommended dose of bosutinib administered orall...

The main purpose of this study is to demonstrate that the growth of infants fed with the Test Formula is non-inferior those fed with the Control Form...

The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric and adult participan...

The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH...

The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH

This is a prospective study for the long-term follow-up (LTFU) of safety and efficacy for all pediatric and adult participants exposed to Gene-modifi...

The purpose of this study is to assess the long-term safety and effectiveness of odevixibat in participants with Alagille syndrome (ALGS).The partici...

This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1...