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 […]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 […]“I just wanted to speak to someone- and there was no one…”: using Burden of Treatment Theory to understand the impact of a novel ATMP on early recipients
Orphanet J Rare Dis. 2023 Apr 17;18(1):86. doi: 10.1186/s13023-023-02680-y. PMID: 37069697; PMCID: PMC10111696. “I just wanted to speak to someone- and there was no one…”: using […]How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis
Orphanet J Rare Dis. 2023 Apr 17;18(1):84. doi: 10.1186/s13023-023-02682-w. PMID: 37069597; PMCID: PMC10108537. How do patients and other members of the public engage with the orphan […]Critical considerations for public engagement in stem cell-related research
Stem Cell Reports. 2023 Feb 14;18(2):420-426. doi: 10.1016/j.stemcr.2023.01.002. Epub 2023 Feb 2. PMID: 36736324; PMCID: PMC10242349. Critical considerations for public engagement in stem cell-related research Authors: […]