Research priorities for liver glycogen storage disease: An international priority setting partnership with the James Lind Alliance.

ABSTRACT: The international liver glycogen storage disease (GSD) priority setting partnership (IGSDPSP) was established to identify the top research priorities in this area. The multiphase methodology followed the principles of the James Lind Alliance (JLA) guidebook. An international scoping survey in seven languages was distributed to patients, carers, and healthcare professionals to gather uncertainties, which were consolidated into summary questions. The existing literature was reviewed to ensure that the summary questions had not yet been answered. A second survey asked responders to prioritize these summary questions. A final shortlist of 22 questions was discussed during an international multi-stakeholder workshop, and a consensus was reached on the top 11 priorities using an adapted nominal

SUBMITTER: Peeks F 

PROVIDER: S-EPMC7079148 | biostudies-literature | 2020 Mar

REPOSITORIES: biostudies-literature