This is a prospective, single-center, randomized, double-blind, placebo-controlled study designed to assess the safety, tolerability, and clinical and metabolic improvement of pediatric subjects with PMM2-CDG on oral epalrestat therapy vs. placebo.

Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG

A Prospective, Randomized, Double-Blind, Placebo-Controlled, Single-Center Study of Oral Epalrestat Therapy in Pediatric Subjects With Phosphomannomutase 2-congenital Disorder of Glycosylation (PMM2-CDG)

Treatment Trials

Focus your search

Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG

Description

Conditions