Clinical Trial Update: A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients [Phase 1]

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Summary

Phase 1, single-stage adaptive open-label dose-escalation study to evaluate safety and initial efficacy of a therapy for Aromatic L-amino acid decarboxylase def…

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Diseases / indications

• Aromatic L-amino Acid Decarboxylase Deficiency

Snippet

Sponsor: Krzysztof Bankiewicz | Conditions: AADC Deficiency | Phase 1 | Status: RECRUITING