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Disease

aromatic L-amino acid decarboxylase deficiency

Also known as AADC deficiency, aromatic L-amino-acid decarboxylase deficiency, Aadc deficiency, DDC deficiency+2 more

AADC deficiency, aromatic L-amino-acid decarboxylase deficiency, Aadc deficiency, DDC deficiency, Dopa decarboxylase deficiency, aromatic amino acid decarboxylase deficiency.

1
Associated genes
1
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

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Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies

Approval — Upstaza is indicated for the treatment of patients aged 18 months and older with a clinic… (2022)

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2022emaApprovalEladocagene exuparvovec· Upstaza is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L amino acid decarboxylase (AADC) deficiency with a severe phenotype (see section 5.1). source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

1 match

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Reference

Authoritative identity, definition & identifiers.

Synonyms

AADC deficiency, aromatic L-amino-acid decarboxylase deficiency, Aadc deficiency, DDC deficiency, Dopa decarboxylase deficiency, aromatic amino acid decarboxylase deficiency

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.