Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease

Inclusion Criteria: * Confirmed diagnosis of APBD by the presence of mutations of the GBE1 gene in both alleles or brancher enzyme deficiency * Willing and able to travel to Dallas TX * Able to tolerate dietary oil * Able to provide informed consent Exclusion Criteria: * Intercurrent medical conditions that would confound the assessment of efficacy, such as HIV or diabetes * Patients who are wheelchair bound * Patients deemed unsuitable for the study by the investigator