Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.
Status:
Completed
Trial end date:
2020-12-30
Target enrollment:
Participant gender:
Summary
In this study phenylbutyrate is used for patients with pyruvate dehydrogenase complex
deficiency. The aim of the study is to investigate the safety and efficacy of therapy.