Ipsen (United Kingdom)

Indirect treatment comparison of odevixibat and maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome

Aim: Alagille syndrome (ALGS) is a rare genetic disorder associated with cholestasis and subsequent severe pruritus, which significantly impacts patient quality of life (QoL). Odevixibat (ODX) and maralixibat (MRX) are ileal bile acid transporter inhibitors approved for the treat...

Clinical efficacy/effectiveness, safety, and health-related quality of life associated with relapsed/refractory pediatric low-grade glioma treatments: systematic literature review

Abstract Background Patients with relapsed/refractory pediatric low-grade glioma (R/R pLGG) face considerable long-term morbidity. Most cases are driven by mitogen-activated protein kinase (MAPK) pathway alterations, for which several targeted agents have been evaluated. Systemat...