Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.

ABSTRACT:

Background

Mucopolysaccharidosis type III is a progressive, neurodegenerative lysosomal storage disorder for which there is currently no effective therapy. Though numerous potential therapies are in development, there are several challenges to conducting clinical research in this area. We seek to make recommendations on the approach to clinical research in MPS III, including the selection of outcome measures and trial endpoints, in order to improve the quality and impact of research in this area.

Results

An international workshop involving academic researchers, clinical experts and industry groups was held in June 2015, with presentations and discussions on disease pathophysiology, biomarkers, potential therapies and clinical outcome measures. A set of recommendations was su

SUBMITTER: Ghosh A 

PROVIDER: S-EPMC5485703 | biostudies-literature | 2017 Jun

REPOSITORIES: biostudies-literature