Selective Serotonin Reuptake Inhibitor Treatment Post Gene Therapy for an Ultrarare Neurometabolic Disorder (AADC Deficiency).
J Am Acad Child Adolesc Psychiatry
; 63(6): 571-573, 2024 Jun.
Article
en En
| MEDLINE
| ID: mdl-38460745
ABSTRACT
A 7-year-old girl presented with persistent anxiety symptoms for several years following gene therapy for an ultrarare neurometabolic disorder (aromatic L-amino acid decarboxylase [AADC] deficiency). AADC is the final enzyme in the monoamine synthesis pathway (Figure 1).1 Its absence results in a severe combined deficiency in serotonin, dopamine, epinephrine, and norepinephrine, causing significant developmental delays, hypotonia, and dystonia. The incidence of AADC deficiency is estimated at â¼1 in 500,000,2 and â¼200 cases have been described.1 Recently available disease-modifying gene therapy for this condition dramatically improves motor symptoms, and received regulatory approval in some regions in 2022.2 There are no data to guide psychiatric care post gene therapy for AADC or other neurologic disorders to date.3.
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Descarboxilasas de Aminoácido-L-Aromático
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Terapia Genética
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Inhibidores Selectivos de la Recaptación de Serotonina
Límite:
Child
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Female
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Humans
Idioma:
En
Revista:
J Am Acad Child Adolesc Psychiatry
Asunto de la revista:
PEDIATRIA
/
PSIQUIATRIA
Año:
2024
Tipo del documento:
Article