Patient engagement in the design of clinical research in Noonan syndrome spectrum disorders: a scoping review.

ABSTRACT:

Background

Noonan syndrome spectrum disorders are a group of disorders caused by mutations in several genes of the RAS/MAPK pathway. Because of a highly heterogeneity and variable phenotypical manifestations of the disorders, these children and adults have a variable number of symptoms. Inclusion of their perceived experience of their health and developmental problems in research (design) could contribute to increased relevance of the research process and outcomes. The aim of this study is to get insight in what way patients with a Noonan syndrome spectrum disorder have been involved in the research process in order to learn for future engagement practices.

Methods and results

To that end, the degree of engagement was measured by the eight levels of the participation ladder

SUBMITTER: Tiemens DK 

PROVIDER: S-EPMC8549341 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature