Bh4 Deficiency

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CLINICAL_TRIALS trial_result · 2023-11-14
Clinical Trial Update: A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia [Phase 1, Phase 2]
Phase 1/2 trial of PTC923 (CNSA-001) in participants with primary BH4 deficiency and hyperphenylalaninemia has completed with results posted…