Matches in SemOpenAlex for { <https://semopenalex.org/work/W2977525204> ?p ?o ?g. }
Showing items 1 to 75 of
75
with 100 items per page.
- W2977525204 abstract "Rare diseases are diseases with prevalence of lower than one in 2,000 individuals. Jointly, rare diseases affect many people in the world. Research in rare diseases is more challenging than in diseases that are more prevalent, due to small numbers, heterogeneity and often limited knowledge about the disease course and the pathophysiology underlying the disease. This thesis has focused on two aspects of clinical research in rare diseases. In part one, patient involvement in rare disease research is investigated. Together with a group of rare disease patient representatives, we have developed a tool in which patient representatives together with researchers decide which outcome measures are relevant to patients, and can be added in a clinical trial. In part two, we have investigated a measurement instrument: Goal Attainment Scaling (GAS). GAS is an assessment instrument that is intended for individual evaluation of an intervention. It allows patients to set individual goals, together with their treating professional. The number of goals and the content of these goals may differ per patient, but the attainment of the goals is measured in a standardized way. This makes a standardized evaluation of an intervention possible, even when the patients are all in a different stage of their disease. This can be an advantage in rare disease research, where the population is often small and heterogeneous. We conclude that more research on the validation of GAS is needed, and propose how GAS can be used and validated in the setting of rare disease trials." @default.
- W2977525204 created "2019-10-10" @default.
- W2977525204 creator A5050107651 @default.
- W2977525204 date "2019-01-01" @default.
- W2977525204 modified "2023-09-26" @default.
- W2977525204 title "Patient involvement in rare disease trial design: Small populations making a big difference" @default.
- W2977525204 hasPublicationYear "2019" @default.
- W2977525204 type Work @default.
- W2977525204 sameAs 2977525204 @default.
- W2977525204 citedByCount "0" @default.
- W2977525204 crossrefType "journal-article" @default.
- W2977525204 hasAuthorship W2977525204A5050107651 @default.
- W2977525204 hasConcept C118552586 @default.
- W2977525204 hasConcept C121332964 @default.
- W2977525204 hasConcept C142724271 @default.
- W2977525204 hasConcept C15744967 @default.
- W2977525204 hasConcept C177264268 @default.
- W2977525204 hasConcept C177713679 @default.
- W2977525204 hasConcept C184779094 @default.
- W2977525204 hasConcept C199360897 @default.
- W2977525204 hasConcept C2779134260 @default.
- W2977525204 hasConcept C2779701055 @default.
- W2977525204 hasConcept C2780218827 @default.
- W2977525204 hasConcept C2780665704 @default.
- W2977525204 hasConcept C2908647359 @default.
- W2977525204 hasConcept C2994388031 @default.
- W2977525204 hasConcept C41008148 @default.
- W2977525204 hasConcept C535046627 @default.
- W2977525204 hasConcept C71924100 @default.
- W2977525204 hasConcept C99454951 @default.
- W2977525204 hasConceptScore W2977525204C118552586 @default.
- W2977525204 hasConceptScore W2977525204C121332964 @default.
- W2977525204 hasConceptScore W2977525204C142724271 @default.
- W2977525204 hasConceptScore W2977525204C15744967 @default.
- W2977525204 hasConceptScore W2977525204C177264268 @default.
- W2977525204 hasConceptScore W2977525204C177713679 @default.
- W2977525204 hasConceptScore W2977525204C184779094 @default.
- W2977525204 hasConceptScore W2977525204C199360897 @default.
- W2977525204 hasConceptScore W2977525204C2779134260 @default.
- W2977525204 hasConceptScore W2977525204C2779701055 @default.
- W2977525204 hasConceptScore W2977525204C2780218827 @default.
- W2977525204 hasConceptScore W2977525204C2780665704 @default.
- W2977525204 hasConceptScore W2977525204C2908647359 @default.
- W2977525204 hasConceptScore W2977525204C2994388031 @default.
- W2977525204 hasConceptScore W2977525204C41008148 @default.
- W2977525204 hasConceptScore W2977525204C535046627 @default.
- W2977525204 hasConceptScore W2977525204C71924100 @default.
- W2977525204 hasConceptScore W2977525204C99454951 @default.
- W2977525204 hasLocation W29775252041 @default.
- W2977525204 hasOpenAccess W2977525204 @default.
- W2977525204 hasPrimaryLocation W29775252041 @default.
- W2977525204 hasRelatedWork W109669460 @default.
- W2977525204 hasRelatedWork W1503184271 @default.
- W2977525204 hasRelatedWork W1596322973 @default.
- W2977525204 hasRelatedWork W1722016699 @default.
- W2977525204 hasRelatedWork W1980553424 @default.
- W2977525204 hasRelatedWork W1993252336 @default.
- W2977525204 hasRelatedWork W2033517762 @default.
- W2977525204 hasRelatedWork W2042390728 @default.
- W2977525204 hasRelatedWork W2048934331 @default.
- W2977525204 hasRelatedWork W2138143000 @default.
- W2977525204 hasRelatedWork W2415922995 @default.
- W2977525204 hasRelatedWork W2416034081 @default.
- W2977525204 hasRelatedWork W2465773512 @default.
- W2977525204 hasRelatedWork W258189349 @default.
- W2977525204 hasRelatedWork W2790153693 @default.
- W2977525204 hasRelatedWork W2890454147 @default.
- W2977525204 hasRelatedWork W2998775105 @default.
- W2977525204 hasRelatedWork W3031628127 @default.
- W2977525204 hasRelatedWork W3049127498 @default.
- W2977525204 hasRelatedWork W3087122444 @default.
- W2977525204 isParatext "false" @default.
- W2977525204 isRetracted "false" @default.
- W2977525204 magId "2977525204" @default.
- W2977525204 workType "article" @default.