139 related articles for article (PubMed ID: 37402126)
1. Gene therapy for aromatic L-amino acid decarboxylase deficiency: Requirements for safe application and knowledge-generating follow-up.
Roubertie A; Opladen T; Brennenstuhl H; Kuseyri Hübschmann O; Flint L; Willemsen MA; Leuzzi V; Cazorla AG; Kurian MA; François-Heude MC; Hwu P; Zeev BB; Kiening K; Roujeau T; Pons R; Pearson TS
J Inherit Metab Dis; 2024 May; 47(3):463-475. PubMed ID: 37402126
[TBL] [Abstract][Full Text] [Related]
2. Eladocagene Exuparvovec: First Approval.
Keam SJ
Drugs; 2022 Sep; 82(13):1427-1432. PubMed ID: 36103022
[TBL] [Abstract][Full Text] [Related]
3. Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency.
Tai CH; Lee NC; Chien YH; Byrne BJ; Muramatsu SI; Tseng SH; Hwu WL
Mol Ther; 2022 Feb; 30(2):509-518. PubMed ID: 34763085
[TBL] [Abstract][Full Text] [Related]
4. Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.
Pearson TS; Gupta N; San Sebastian W; Imamura-Ching J; Viehoever A; Grijalvo-Perez A; Fay AJ; Seth N; Lundy SM; Seo Y; Pampaloni M; Hyland K; Smith E; de Oliveira Barbosa G; Heathcock JC; Minnema A; Lonser R; Elder JB; Leonard J; Larson P; Bankiewicz KS
Nat Commun; 2021 Jul; 12(1):4251. PubMed ID: 34253733
[TBL] [Abstract][Full Text] [Related]
5. Consensus guideline for the diagnosis and treatment of aromatic l-amino acid decarboxylase (AADC) deficiency.
Wassenberg T; Molero-Luis M; Jeltsch K; Hoffmann GF; Assmann B; Blau N; Garcia-Cazorla A; Artuch R; Pons R; Pearson TS; Leuzzi V; Mastrangelo M; Pearl PL; Lee WT; Kurian MA; Heales S; Flint L; Verbeek M; Willemsen M; Opladen T
Orphanet J Rare Dis; 2017 Jan; 12(1):12. PubMed ID: 28100251
[TBL] [Abstract][Full Text] [Related]
6. A position statement on the post gene-therapy rehabilitation of aromatic I-amino acid decarboxylase deficiency patients.
Lee HM; Mercimek-Andrews S; Horvath G; Marchese D; Poulin RE; Krolick A; Tierney KL; Turna J; Wei J; Hwu WL
Orphanet J Rare Dis; 2024 Jan; 19(1):17. PubMed ID: 38238766
[TBL] [Abstract][Full Text] [Related]
7. Long-Term Outcomes of Eladocagene Exuparvovec Compared with Standard of Care in Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency: A Modelling Study.
Simons CL; Hwu WL; Zhang R; Simons MJHG; Bergkvist M; Bennison C
Adv Ther; 2023 Dec; 40(12):5399-5414. PubMed ID: 37803205
[TBL] [Abstract][Full Text] [Related]
8. Aromatic amino acid decarboxylase deficiency: Molecular and metabolic basis and therapeutic outlook.
Himmelreich N; Montioli R; Bertoldi M; Carducci C; Leuzzi V; Gemperle C; Berner T; Hyland K; Thöny B; Hoffmann GF; Voltattorni CB; Blau N
Mol Genet Metab; 2019 May; 127(1):12-22. PubMed ID: 30952622
[TBL] [Abstract][Full Text] [Related]
9. A review of aromatic l-amino acid decarboxylase (AADC) deficiency in Taiwan.
Lee NC; Chien YH; Hwu WL
Am J Med Genet C Semin Med Genet; 2019 Jun; 181(2):226-229. PubMed ID: 30614627
[TBL] [Abstract][Full Text] [Related]
10. Newborn screening for aromatic l-amino acid decarboxylase deficiency - Strategies, results, and implication for prevalence calculations.
Reischl-Hajiabadi AT; Okun JG; Kohlmüller D; Manukjan G; Hegert S; Durner J; Schuhmann E; Hörster F; Mütze U; Feyh P; Hoffmann GF; Röschinger W; Janzen N; Opladen T
Mol Genet Metab; 2024 Mar; 141(3):108148. PubMed ID: 38302374
[TBL] [Abstract][Full Text] [Related]
11. Prevalence of Aromatic l-Amino Acid Decarboxylase Deficiency in At-Risk Populations.
Hyland K; Reott M
Pediatr Neurol; 2020 May; 106():38-42. PubMed ID: 32111562
[TBL] [Abstract][Full Text] [Related]
12. Gene therapy improves motor and mental function of aromatic l-amino acid decarboxylase deficiency.
Kojima K; Nakajima T; Taga N; Miyauchi A; Kato M; Matsumoto A; Ikeda T; Nakamura K; Kubota T; Mizukami H; Ono S; Onuki Y; Sato T; Osaka H; Muramatsu SI; Yamagata T
Brain; 2019 Feb; 142(2):322-333. PubMed ID: 30689738
[TBL] [Abstract][Full Text] [Related]
13. Selective Serotonin Reuptake Inhibitor Treatment Post Gene Therapy for an Ultrarare Neurometabolic Disorder (AADC Deficiency).
Baribeau DA; Vorstman JAS; Pearson TS
J Am Acad Child Adolesc Psychiatry; 2024 Jun; 63(6):571-573. PubMed ID: 38460745
[TBL] [Abstract][Full Text] [Related]
14. A pathogenic S250F missense mutation results in a mouse model of mild aromatic l-amino acid decarboxylase (AADC) deficiency.
Caine C; Shohat M; Kim JK; Nakanishi K; Homma S; Mosharov EV; Monani UR
Hum Mol Genet; 2017 Nov; 26(22):4406-4415. PubMed ID: 28973165
[TBL] [Abstract][Full Text] [Related]
15. Semi-quantitative detection of a vanillactic acid/vanillylmandelic acid ratio in urine is a reliable diagnostic marker for aromatic L-amino acid decarboxylase deficiency.
Brennenstuhl H; Garbade SF; Okun JG; Feyh P; Hoffmann GF; Langhans CD; Opladen T
Mol Genet Metab; 2020; 131(1-2):163-170. PubMed ID: 32675002
[TBL] [Abstract][Full Text] [Related]
16. Benefits of Neuronal Preferential Systemic Gene Therapy for Neurotransmitter Deficiency.
Lee NC; Muramatsu S; Chien YH; Liu WS; Wang WH; Cheng CH; Hu MK; Chen PW; Tzen KY; Byrne BJ; Hwu WL
Mol Ther; 2015 Oct; 23(10):1572-81. PubMed ID: 26137853
[TBL] [Abstract][Full Text] [Related]
17. Intraputaminal Gene Delivery in Two Patients with Aromatic L-Amino Acid Decarboxylase Deficiency.
François-Heude MC; Poulen G; Flamand Roze E; Nguyen Morel MA; Gras D; Roch-Toreilles I; Quintard A; Baroux G; Meyer P; Coubes P; Milesi C; Cambonie G; Baleine J; Sola C; Delye B; Dimopoulou E; Sanchez S; Gasnier M; Touati S; Zamora A; Pontal D; Leboucq N; Kouyoumdjian V; Lebasnier A; Sanquer S; Mariano-Goulart D; Roujeau T; Roubertie A
Mov Disord Clin Pract; 2023 May; 10(5):811-818. PubMed ID: 37205256
[TBL] [Abstract][Full Text] [Related]
18. Detection of 3-O-methyldopa in dried blood spots for neonatal diagnosis of aromatic L-amino-acid decarboxylase deficiency: The northeastern Italian experience.
Burlina A; Giuliani A; Polo G; Gueraldi D; Gragnaniello V; Cazzorla C; Opladen T; Hoffmann G; Blau N; Burlina AP
Mol Genet Metab; 2021 May; 133(1):56-62. PubMed ID: 33744095
[TBL] [Abstract][Full Text] [Related]
19. [Anesthetic Care of Children With Gene Therapy for the Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency].
Li WC; Fan SZ
Hu Li Za Zhi; 2019 Feb; 66(1):107-114. PubMed ID: 30648251
[TBL] [Abstract][Full Text] [Related]
20. Gene Therapy for Dopamine Dyshomeostasis: From Parkinson's to Primary Neurotransmitter Diseases.
Ng J; Barral S; Waddington SN; Kurian MA
Mov Disord; 2023 Jun; 38(6):924-936. PubMed ID: 37147851
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]