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First received: April 04, 2023

Clinical Trial: Orphan Drugs for Inherited Metabolic Diseases

Study Status: Completed

Recruit Status: RECRUITING

Condition: Inborn Errors of Metabolism

Study Type: OBSERVATIONAL


Official Title: The Challenge of Treating Orphan Diseases With Novel Therapies: Experience From One Adult Metabolic Clinic in Switzerland

Brief Summary: The aim of this study is to report and describe all the patients with confirmed diagnosis of inherited metabolic disease (IMD) treated with orphan medicinal products (OMPs) in a cohort of adult patients followed in a reference center for rare diseases (Lausanne University Hospital, CHUV) from 2017-2022.

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