Development of a core outcome set for the evaluation of treatments for Guanidinoacetate Methyltransferase (GAMT) Deficiency: A Rapid Review

Autor: Moghaddam, Zahra, Li, Jasmine, Stockler, Sylvia, Apatean, Delia
Rok vydání: 2022
Předmět:
DOI: 10.17605/osf.io/jau7v
Popis: This is a rapid review regarding the outcomes and symptoms associated with GAMT deficiency reported in the literature, with the goal of developing a list of core outcome sets for treatment of cerebral creatine deficiencies (in conjunction with another literature review about Creatine transporter deficiency and focus groups conducted with parents of children with GAMT deficiency and CTD)
Databáze: OpenAIRE