Clinical trials: Genetic of Intellectual Deficiency
Pyruvate Dehydrogenase Complex Deficiency
Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.
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.
- Trial name
- TIGEM2-PDH (NCT03734263)
- Testing
- sodium phenylbutyrate
- Where
- Italy
- Volunteers
- 1 people
- Sponsor
- Fondazione Telethon