Efficacy Confirmation Study of NPC-09

Authors
Category Primary study
Registry of Trialsclinicaltrials.gov
Year 2021
GNE myopathy is a distal myopathy that is thought to be caused by a mutation in the GNE gene that encodes an enzyme in the biosynthetic process of aceneuramic acid (typical sialic acid). The investigators will examine the efficacy and safety of aceneuramic acid (SA-ER tablets) 6g daily for 48 weeks in patients with GNE myopathy in a placebo-controlled, double-blind, controlled trial.
Epistemonikos ID: 190c564dce960be98d6d814bd23eeb4d160ea8d7
First added on: May 08, 2024