Aromatic L-amino Acid Decarboxylase Deficiency
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CLINICAL_TRIALS research · 2026-04-21Clinical Trial Update: A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency [Phase 2, Phase 3]
The document reports an ongoing Phase 2/Phase 3 clinical trial of VGN-R09b for Aromatic L-amino Acid Decarboxylase Deficiency, with the stud…
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CLINICAL_TRIALS research · 2025-10-21Clinical Trial Update: A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients [Phase 1]
Phase 1, single-stage adaptive open-label dose-escalation study to evaluate safety and initial efficacy of a therapy for Aromatic L-amino ac…
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CLINICAL_TRIALS trial_result · 2023-02-10Clinical Trial Update: A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC [Phase 1, Phase 2]
Phase I/II clinical trial evaluating AAV2-hAADC gene therapy for Aromatic L-amino Acid Decarboxylase (AADC) deficiency has completed.
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EMA approval · 2022-05-20First therapy to treat rare genetic nervous system disorder AADC deficiency
The European Medicines Agency announces the first therapy approved to treat AADC deficiency, a rare genetic nervous system disorder.
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CLINICAL_TRIALS trial_result · 2017-04-28Clinical Trial Update: Newborn Screening for Aromatic L-amino Acid Decarboxylase Deficiency
A completed clinical trial assessing newborn screening for aromatic L-amino acid decarboxylase deficiency in newborns.
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