Clinical Trial Update: A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia [Phase 1, Phase 2]

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Summary

Phase 1/2 trial of PTC923 (CNSA-001) in participants with primary BH4 deficiency and hyperphenylalaninemia has completed with results posted.

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Diseases / indications

• Bh4 Deficiency
• Hyperphenylalaninemia

Drugs mentioned

• PTC923 (CNSA-001) (PTC923)

Snippet

Sponsor: PTC Therapeutics | Conditions: BH4 Deficiency, Hyperphenylalaninemia | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2023-11-14