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Phase 2 Study of HST5040 in Propionic Acidemia and Methylmalonic Acidemia
2023-000785-33Phase 2
- Sponsor
- HemoShear Therapeutics
- Start
- Aug 2023
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, randomised, double-blind trial of sodium 2,2-dimethylbutyrate in propionic acidemia (PA) and Methylmalonic acidemia mutase type (MMA) (4 arms), sponsored by HemoShear Therapeutics. Primary endpoint: Safety of HST5040 in PA and MMA (Part A).
02
Drugs
sodium 2,2-dimethylbutyrate
03
Conditions
propionic acidemia (PA)Methylmalonic acidemia mutase type (MMA)
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