A double‐blind, placebo‐controlled trial of triheptanoin in adult polyglucosan body disease and open‐label, long‐term outcome

Abstract

We cannot conclude that triheptanoin was effective in the treatment of APBD over a 6-month period, but we found it had a good safety profile. This study also emphasizes the difficulty of conducting trials in very rare diseases presenting with a wide clinical heterogeneity. ClinicalTrials.gov Identifier: NCT00947960.