Selective Serotonin Reuptake Inhibitor Treatment Post Gene Therapy for an Ultrarare Neurometabolic Disorder (AADC Deficiency).
| Autor: | Baribeau DA; Holland Bloorview Kids Rehabilitation Hospital, Toronto, Ontario, Canada; The Hospital for Sick Children, Toronto, Ontario, Canada. Electronic address: danielle.baribeau@mail.utoronto.ca., Vorstman JAS; The Hospital for Sick Children, Toronto, Ontario, Canada., Pearson TS; Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio. |
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| Jazyk: | angličtina |
| Zdroj: | Journal of the American Academy of Child and Adolescent Psychiatry [J Am Acad Child Adolesc Psychiatry] 2024 Jun; Vol. 63 (6), pp. 571-573. Date of Electronic Publication: 2024 Mar 07. |
| DOI: | 10.1016/j.jaac.2024.01.015 |
| Abstrakt: | 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 ∼1 in 500,000, 2 and ∼200 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 . (Copyright © 2024 American Academy of Child and Adolescent Psychiatry. Published by Elsevier Inc. All rights reserved.) |
| Databáze: | MEDLINE |
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