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 |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 42152020 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: Access to orphan drugs in adults with inherited metabolic diseases in Switzerland: a single-center retrospective cohort study. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Antoni+G%22">Antoni G</searchLink>; Division of Genetic Medicine, University of Lausanne and University Hospital of Lausanne, Lausanne, Switzerland.<br /><searchLink fieldCode="AU" term="%22Kumps+C%22">Kumps C</searchLink>; Division of Genetic Medicine, University of Lausanne and University Hospital of Lausanne, Lausanne, Switzerland.<br /><searchLink fieldCode="AU" term="%22Berger+J%22">Berger J</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Barman-Aksözen+J%22">Barman-Aksözen J</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Tran+C%22">Tran C</searchLink>; Division of Genetic Medicine, University of Lausanne and University Hospital of Lausanne, Lausanne, Switzerland. christel.tran@chuv.ch. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%22101266602%22">Orphanet journal of rare diseases</searchLink> [Orphanet J Rare Dis] 2026 May 18; Vol. 21 (1). <i>Date of Electronic Publication: </i>2026 May 18. – Name: TypePub Label: Publication Type Group: TypPub Data: Journal Article – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22BioMed+Central%22">BioMed Central </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>101266602 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1750-1172 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2217501172%22">17501172 </searchLink><i>NLM ISO Abbreviation: </i>Orphanet J Rare Dis <i>Subsets: </i>MEDLINE |
| PLink | https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=42152020 |
| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1186/s13023-026-04367-6 Languages: – Code: eng Text: English Titles: – TitleFull: Access to orphan drugs in adults with inherited metabolic diseases in Switzerland: a single-center retrospective cohort study. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Antoni G – PersonEntity: Name: NameFull: Kumps C – PersonEntity: Name: NameFull: Berger J – PersonEntity: Name: NameFull: Barman-Aksözen J – PersonEntity: Name: NameFull: Tran C IsPartOfRelationships: – BibEntity: Dates: – D: 18 M: 05 Text: 2026 May 18 Type: published Y: 2026 Identifiers: – Type: issn-electronic Value: 1750-1172 Numbering: – Type: volume Value: 21 – Type: issue Value: 1 Titles: – TitleFull: Orphanet journal of rare diseases Type: main |
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