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Orphan Drugs for Inherited Metabolic Diseases

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  • المصدر:
    The Challenge of Treating Orphan Diseases With Novel Therapies: Experience From One Adult Metabolic Clinic in Switzerland
    Gariani K, Nascimento M, Superti-Furga A, Tran C. Clouds over IMD? Perspectives for inherited metabolic diseases in adults from a retrospective cohort study in two Swiss adult metabolic clinics. Orphanet J Rare Dis. 2020 Aug 18;15(1):210. doi: 10.1186/s13023-020-01471-z.