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1.
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial.
Mol Ther
; 32(3): 609-618, 2024 Mar 06.
Article
in English
| MEDLINE | ID: mdl-38204164
2.
Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II: A phase 2 trial in Brazil.
Mol Ther
; 29(7): 2378-2386, 2021 07 07.
Article
in English
| MEDLINE | ID: mdl-33781915
3.
A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II.
Mol Ther
; 29(2): 671-679, 2021 02 03.
Article
in English
| MEDLINE | ID: mdl-33038326
4.
Drug delivery for neuronopathic lysosomal storage diseases: evolving roles of the blood brain barrier and cerebrospinal fluid.
Metab Brain Dis
; 37(6): 1745-1756, 2022 08.
Article
in English
| MEDLINE | ID: mdl-35088290
5.
Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis of Preclinical and Clinical Data.
Int J Mol Sci
; 22(20)2021 Oct 10.
Article
in English
| MEDLINE | ID: mdl-34681597
6.
Efficacy and safety of GH treatment in Japanese children with short stature due to SHOX deficiency: a randomized phase 3 study.
Clin Pediatr Endocrinol
; 33(2): 43-49, 2024.
Article
in English
| MEDLINE | ID: mdl-38572386
7.
Analysis of caregiver perspectives on patients with mucopolysaccharidosis II treated with pabinafusp alfa: results of qualitative interviews in Japan.
Orphanet J Rare Dis
; 19(1): 104, 2024 Mar 07.
Article
in English
| MEDLINE | ID: mdl-38454486
8.
Treatment of Neuronopathic Mucopolysaccharidoses with Blood-Brain Barrier-Crossing Enzymes: Clinical Application of Receptor-Mediated Transcytosis.
Pharmaceutics
; 14(6)2022 Jun 11.
Article
in English
| MEDLINE | ID: mdl-35745811
9.
Pharmacokinetics and pharmacodynamics of once-weekly administration of JR-142, a long-acting albumin-fused human growth hormone: A rondemized, placebo-controlled phase 1 study.
Growth Horm IGF Res
; 67: 101500, 2022 Dec.
Article
in English
| MEDLINE | ID: mdl-36113378
10.
Divergent developmental trajectories in two siblings with neuropathic mucopolysaccharidosis type II (Hunter syndrome) receiving conventional and novel enzyme replacement therapies: A case report.
JIMD Rep
; 62(1): 9-14, 2021 Nov.
Article
in English
| MEDLINE | ID: mdl-34765392
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