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Phenylbutyrate Therapy in Mitochondrial Disease with Lactic Acidosis (MELAS and PDH deficiency)
2018-001094-25Phase 2Completed
- Sponsor
- Carlo Besta Neurological Institute
- Start
- May 2021
- Modality
- Small Molecules
- Countries
- 1
01
Overview
A Phase 2 trial of sodium phenylbutyrate in Mitochondriopathy, mitochondrial encephalomyopathy lactic acidosis and stroke-like episodes (MELAS), pyruvate dehydrogenase deficiency (PDH), sponsored by Carlo Besta Neurological Institute. Started May 2021; status: completed.
02
Drugs
sodium phenylbutyrate
03
Conditions
Mitochondriopathymitochondrial encephalomyopathy lactic acidosis and stroke-like episodes (MELAS)pyruvate dehydrogenase deficiency (PDH)
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