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

ConclusionsWe 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.
Source: Journal of Inherited Metabolic Disease - Category: Internal Medicine Source Type: research