Access to orphan drugs in adults with inherited metabolic diseases in Switzerland: a single-center retrospective cohort study.
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| Title: | Access to orphan drugs in adults with inherited metabolic diseases in Switzerland: a single-center retrospective cohort study. |
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| Authors: | Antoni G; Division of Genetic Medicine, University of Lausanne and University Hospital of Lausanne, Lausanne, Switzerland., Kumps C; Division of Genetic Medicine, University of Lausanne and University Hospital of Lausanne, Lausanne, Switzerland., Berger J; Community Pharmacy, Center for Primary Care and Public Health (Unisanté), University of Lausanne, Lausanne, Switzerland.; Center for Research and Innovation in Clinical Pharmaceutical Sciences, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland.; School of Pharmaceutical Sciences, University of Geneva, Geneva, Switzerland.; Institute of Pharmaceutical Sciences of Western Switzerland, University of Geneva and University of Lausanne, Geneva and Lausanne, Switzerland., Barman-Aksözen J; Institute of Laboratory Medicine, Municipal Hospital Zurich Triemli, Zurich, Switzerland.; Division of Metabolism and Children's Research Center, University Children's Hospital, Zurich, Switzerland.; University Research Priority Program 'ITINERARE - Innovative Therapies in Rare Diseases', University of Zurich, Zurich, Switzerland., Tran C; Division of Genetic Medicine, University of Lausanne and University Hospital of Lausanne, Lausanne, Switzerland. christel.tran@chuv.ch. |
| Source: | Orphanet journal of rare diseases [Orphanet J Rare Dis] 2026 May 18; Vol. 21 (1). Date of Electronic Publication: 2026 May 18. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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