Matches in SemOpenAlex for { <https://semopenalex.org/work/W4313888051> ?p ?o ?g. }
Showing items 1 to 45 of
45
with 100 items per page.
- W4313888051 endingPage "167" @default.
- W4313888051 startingPage "117" @default.
- W4313888051 abstract "According to National Institutes of Health, there are approximately 7,000 rare diseases affecting between 25 and 30 million Americans. Among those, approximately 50% of the people affected by rare diseases are children. Children can benefit tremendously from new therapies, especially when the rare diseases are due to genetic deficiency, to live a longer life with quality. In this chapter, various aspects in clinical development in pediatric rare diseases are discussed, including review of global regulations and regulatory programs that promote rare disease and pediatric drug development, the role of natural history and patient advocacy, the applicability of preclinical and nonclinical models, considerations of pediatric extrapolation, the study designs when no or partial extrapolation is considered, key approaches and considerations in use of modeling and simulations, useful Bayesian approaches, and other relevant considerations." @default.
- W4313888051 created "2023-01-10" @default.
- W4313888051 creator A5007012328 @default.
- W4313888051 creator A5010971048 @default.
- W4313888051 creator A5015176786 @default.
- W4313888051 creator A5030061648 @default.
- W4313888051 creator A5040776117 @default.
- W4313888051 creator A5061433110 @default.
- W4313888051 creator A5066044982 @default.
- W4313888051 date "2023-01-09" @default.
- W4313888051 modified "2023-09-23" @default.
- W4313888051 title "Clinical Development of Pediatric Program within Rare Diseases" @default.
- W4313888051 doi "https://doi.org/10.1201/9781003080954-6" @default.
- W4313888051 hasPublicationYear "2023" @default.
- W4313888051 type Work @default.
- W4313888051 citedByCount "1" @default.
- W4313888051 countsByYear W43138880512023 @default.
- W4313888051 crossrefType "book-chapter" @default.
- W4313888051 hasAuthorship W4313888051A5007012328 @default.
- W4313888051 hasAuthorship W4313888051A5010971048 @default.
- W4313888051 hasAuthorship W4313888051A5015176786 @default.
- W4313888051 hasAuthorship W4313888051A5030061648 @default.
- W4313888051 hasAuthorship W4313888051A5040776117 @default.
- W4313888051 hasAuthorship W4313888051A5061433110 @default.
- W4313888051 hasAuthorship W4313888051A5066044982 @default.
- W4313888051 hasConcept C71924100 @default.
- W4313888051 hasConceptScore W4313888051C71924100 @default.
- W4313888051 hasLocation W43138880511 @default.
- W4313888051 hasOpenAccess W4313888051 @default.
- W4313888051 hasPrimaryLocation W43138880511 @default.
- W4313888051 hasRelatedWork W1506200166 @default.
- W4313888051 hasRelatedWork W1995515455 @default.
- W4313888051 hasRelatedWork W2048182022 @default.
- W4313888051 hasRelatedWork W2080531066 @default.
- W4313888051 hasRelatedWork W2604872355 @default.
- W4313888051 hasRelatedWork W2748952813 @default.
- W4313888051 hasRelatedWork W2899084033 @default.
- W4313888051 hasRelatedWork W3031052312 @default.
- W4313888051 hasRelatedWork W3032375762 @default.
- W4313888051 hasRelatedWork W3108674512 @default.
- W4313888051 isParatext "false" @default.
- W4313888051 isRetracted "false" @default.
- W4313888051 workType "book-chapter" @default.