Efficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan.

ABSTRACT:

Background

A rare muscle disease, GNE myopathy is caused by mutations in the GNE gene involved in sialic acid biosynthesis. Our recent phase II/III study has indicated that oral administration of aceneuramic acid to patients slows disease progression.

Methods

We conducted a phase III, randomized, placebo-controlled, double-blind, parallel-group, multicenter study. Participants were assigned to receive an extended-release formulation of aceneuramic acid (SA-ER) or placebo. Changes in muscle strength and function over 48 weeks were compared between treatment groups using change in the upper extremity composite (UEC) score from baseline to Week 48 as the primary endpoint and the investigator-assessed efficacy rate as the key secondary endpoint. For safety, adverse events, vital

SUBMITTER: Mori-Yoshimura M 

PROVIDER: S-EPMC10416530 | biostudies-literature | 2023 Aug

REPOSITORIES: biostudies-literature