Articles
Patient pathways for rare diseases in Europe: ataxia as an example
Orphanet J Rare Dis 18, 328 (2023). https://doi.org/10.1186/s13023-023-02907-y Patient pathways for rare diseases in Europe: ataxia as an example Authors: Julie Vallortigara1, Julie Greenfield2, Barry Hunt2, Deborah […]Parental Intervention Program for Preschool children with Rare Diseases – a mixed methods evaluation of parents’ experiences and utility
Orphanet J Rare Dis. 2023 Oct 17;18(1):327. doi: 10.1186/s13023-023-02935-8. PMID: 37848938. Parental Intervention Program for Preschool children with Rare Diseases – a mixed methods evaluation of […]Pharmacovigilance for rare diseases: a bibliometrics and knowledge-map analysis based on web of science
Orphanet J Rare Dis. 2023 Sep 26;18(1):303. doi: 10.1186/s13023-023-02915-y. PMID: 37752556. Pharmacovigilance for rare diseases: a bibliometrics and knowledge-map analysis based on web of science Authors: […]ORPHAcodes use for the coding of rare diseases: comparison of the accuracy and cross country comparability
Orphanet J Rare Dis. 2023 Sep 4;18(1):267. doi: 10.1186/s13023-023-02864-6. PMID: 37667299. ORPHAcodes use for the coding of rare diseases: comparison of the accuracy and cross country […]Composite endpoints, including patient reported outcomes, in rare diseases
Orphanet J Rare Dis. 2023 Sep 1;18(1):262. doi: 10.1186/s13023-023-02819-x. PMID: 37658423; PMCID: PMC10474650. Composite endpoints, including patient reported outcomes, in rare diseases Authors: Johan Verbeeck 1, Maya Dirani 2, Johann […]Overview of patients’ cohorts in the French National rare disease registry
Keywords: Rare disease national registry, Fnaace