Endocrinology Trials

• 30/06/2021

  ORION-3

  An open label active comparator extension trial to assess the effect of long term dosing of inclisiran and evolocumab given as subcutaneous injections in subjects with high cardiovascular risk and elevated LDL-C (ORION-3)

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• 30/09/2019

  GalNET

  European registry and network for Galactosemias (GalNET)

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• 31/10/2019

  Dietary management clinical status and outcome of patients with Citrin deficiency in the UK

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• 30/04/2022

  A Comparison of the Natural History of Patients with and without Familial Chylomicronaemia Syndrome (FCS).

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• 31/01/2020

  Blood phenylalanine control of patients with Phenylketonuria in the UK

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• 31/01/2021

  A Phase 2 Randomized Placebo-Controlled Study of Safety and Efficacy Following Repeat-Dose Administration of Evinacumab (anti-ANGPTL3) in Patients with Severe Hypertriglyceridemia (sHTG) at Risk for Acute Pancreatitis

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• 29/06/2018

  Development of health-related quality of life scales for patients and parents affected by Niemann-Pick Disease Type C

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• 01/05/2025

  MARS

  MORQUIO A REGISTRY STUDY(MARS)

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• 18/11/2015

  PKU Lophlex New Format – acceptability and tolerance (market research)

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• 31/12/2019

  An Open-Label Rollover Study of PBI-4050 in Subjects with Alström Syndrome

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