Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI)

Recruiting

Phase 2 — Testing in a larger group (up to a few hundred people) to further evaluate safety and begin to assess effectiveness.

Trial locations(1 site)

France

Hôpital Universitaire Necker - Enfants Malades, Paris, France