Improving clinical trial readiness to accelerate development of new therapeutics for Rett syndrome.

ABSTRACT: Rett syndrome is associated with severe functional impairments and many comorbidities, each in urgent need of treatments. Mutations in the MECP2 gene were identified as causing Rett syndrome in 1999. Over the past 20 years there has been an abundance of preclinical research with some studies leading to human clinical trials. Despite this, few viable therapeutic options have emerged from this investment of effort. Reasons for this lack of success as they relate both to preclinical research and the clinical trial landscape are discussed. Considering what needs to be done to promote further success in the field, we take a positive and constructive approach and introduce the concept of clinical trial readiness and its necessary ingredients for Rett syndrome. These include: listening to the nee

SUBMITTER: Leonard H 

PROVIDER: S-EPMC8894842 | biostudies-literature | 2022 Mar

REPOSITORIES: biostudies-literature