Detalhe da pesquisa
1.
Clinical effect and safety profile of pegzilarginase in patients with arginase 1 deficiency.
J Inherit Metab Dis
; 44(4): 847-856, 2021 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-33325055
2.
Final results of the phase 1/2, open-label clinical study of intravenous recombinant human N-acetyl-α-d-glucosaminidase (SBC-103) in children with mucopolysaccharidosis IIIB.
Mol Genet Metab
; 126(2): 131-138, 2019 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-30635159
3.
Efficacy and safety of pegzilarginase in arginase 1 deficiency (PEACE): a phase 3, randomized, double-blind, placebo-controlled, multi-centre trial.
EClinicalMedicine
; 68: 102405, 2024 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-38292042
4.
Natural history of arginase 1 deficiency and the unmet needs of patients: A systematic review of case reports.
JIMD Rep
; 63(4): 330-340, 2022 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-35822089
5.
Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III.
Orphanet J Rare Dis
; 12(1): 117, 2017 06 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-28651568