Adaptive and innovative study design in rare diseases.

Autor: Ramanan AV; Department of Paediatric Rheumatology, Bristol Royal Hospital for Children, Bristol, UK avramanan@hotmail.com.; Translational Health Sciences, University of Bristol, Bristol, UK., Wason JMS; Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK.
Jazyk: angličtina
Zdroj: Archives of disease in childhood [Arch Dis Child] 2024 Jun 11. Date of Electronic Publication: 2024 Jun 11.
DOI: 10.1136/archdischild-2024-327143
Abstrakt: Competing Interests: Competing interests: JW—none relevant to the manuscript but funding to institution from AstraZeneca, Boehringer-Ingelheim, Hemay, Mirobio, Nemeysis, Novartis, Orbsen, UCB and Worg. AVR—none relevant to the manuscript; speaker fees/honoraria/consultancy from AbbVie, Eli Lilly, UCB, SOBI, Novartis, AstraZeneca and Roche.
Databáze: MEDLINE