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Disorder of catecholamine synthesis

MONDO:0017759

3 clinical trials for this condition and its sub-types.

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Sub-types

Aromatic L-amino acid decarboxylase deficiency (3) Orthostatic hypotension 1 (0)

Broader categories

Disease (717) Metabolic disease (241) Hereditary disease (188) Inborn errors of metabolism (47) Human disease (15) Disease of genetic or genomic mechanism (2) Disease by developmental or physiological process (0) Disease by etiologic mechanism (0) Inborn disorder of biogenic amine metabolism and transport (0) Inborn disorder of neurotransmitter metabolism and transport (0)
Trials to join now! 1 Not yet finished but already full! 2
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  • Brain gene therapy trial offers hope for kids with rare movement disorder

    Disease control Recruiting now

    This early-stage trial tests a gene therapy called AAV2-hAADC in 42 children with AADC deficiency, a rare genetic disorder that causes severe movement problems and developmental delays. The therapy delivers a working gene directly into brain areas that control movement, aiming to…

    Phase 1 • Sponsor: Krzysztof Bankiewicz • Aim: Disease control

    Last updated Aug 29, 2026 00:00 UTC

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