Results 11 to 20 of about 172,212 (109)

Transferrin Receptor-Targeted Iduronate-2-Sulfatase Penetrates the Blood-Retinal Barrier and Improves Retinopathy in Mucopolysaccharidosis II Mice [PDF]

Molecular Pharmaceutics, 2023
Mucopolysaccharidoses (MPSs) make up a group of lysosomal storage diseases characterized by the aberrant accumulation of glycosaminoglycans throughout the body.
Kohtaro Minami, Hideto Morimoto, Tohru Hirato   +2 more
exaly   +3 more sources

New Paradigms in Management of Central Nervous System Damage in Patients with Hunter Syndrome

Вопросы современной педиатрии
Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is a progressive lysosomal storage disease with X-linked recessive inheritance. This disease is caused by pathogenic or probably pathogenic variants in the IDS gene encoding iduronate 2-sulfatase ...
Nato D. Vashakmadze, Nataly V. Zhurkova, Larisa E. Tsyngunova, Leyla S. Namazova-Baranova   +3 more
doaj   +2 more sources

Additional file 1 of Analysis of caregiver perspectives on patients with mucopolysaccharidosis II treated with pabinafusp alfa: results of qualitative interviews in Japan

Additional Files Supplementary Data for VoC MPSII Treated with Pabinafusp Alfa: includes referenced tables and ...
Nakamura, Kimitoshi, Sakai, Norio, Hossain, Mohammad Arif, Eisengart, Julie B, Yamamoto, Tatsuyoshi, Tanizawa, Kazunori, So, Sairei, Schmidt, Mathias, Sato, Yuji   +8 more
openaire   +2 more sources

Antibodies to watch in 2022

mAbs, 2022
In this 13th annual installment of the annual ‘Antibodies to Watch’ article series, we discuss key events in commercial antibody therapeutics development that occurred in 2021 and forecast events that might occur in 2022.
Hélène Kaplon, Alicia Chenoweth, Silvia Crescioli, Janice M. Reichert   +3 more
doaj   +1 more source

DataSheet2_Transferrin receptor-targeting property of pabinafusp alfa facilitates its uptake by various types of human brain-derived cells in vitro.pdf

, 2023
Pabinafusp alfa, which is an anti-mucopolysaccharidosis II drug, consists of iduronate-2-sulfatase (IDS) genetically fused with an anti-transferrin receptor (TfR) antibody.
Tomomi Furihata, Hiroyuki Sonoda (16482141), Tomoki Fukatsu (16482138), Hanae Morio (12974945), Tomoki Fukatsu, Tomomi Furihata (562810), Hiroyuki Sonoda, Hanae Morio   +7 more
core   +1 more source

Resolution of Extensive Mongolian Spots (Dermal Melanocytosis) in a Patient With Mucopolysaccharidosis Type II Undergoing Enzyme Replacement Therapy With Pabinafusp Alfa: A Case Report

Brain and development. Case reports
Motomichi Kosuga, Tatsuyoshi Yamamoto
exaly   +2 more sources

Caregiver Perspectives on Patients with Mucopolysaccharidosis II Treated with Pabinafusp Alfa: Results of Qualitative Interviews in Japan

, 2023
Abstract Background Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is a rare inherited X-linked metabolic disorder predominantly affecting males. Pabinafusp alfa, an iduronate-2-sulfatase enzyme that can cross the blood-brain barrier, was approved in Japan in 2021 for use in the first enzyme replacement therapy targeting both the
Kimitoshi Nakamura, Norio Sakai, Mohammad Arif Hossain, Julie B. Eisengart, Tatsuyoshi Yamamoto, Kazunori Tanizawa, Sairei So, Mathias Schmidt, Yuji Sato   +8 more
openaire   +1 more source

Pharmacological property, mechanism of action and clinical study results of Pabinafusp Alfa (Genetical Recombination) (IZCARGO^® I.V. Infusion 10 mg) as the therapeutic for Mucopolysaccharidosis type-II (Hunter syndrome)

Folia Pharmacologica Japonica, 2022
Mucopolysaccharidosis type II (MPS II) is an X-linked recessive lysosomal storage disease with the accumulation of glycosaminoglycans in tissues and organs throughout the body caused by dysfunction or loss of iduronate-2-sulfatase (IDS), resulting in somatic and central nervous system (CNS) disorders.
Ryuji, Yamamoto, Satoshi, Kawashima
openaire   +3 more sources

Nanoparticle‐Mediated Therapy for Glioma: Advances and Prospects

MedComm – Oncology, Volume 5, Issue 3, September 2026.
Various nanocarriers, including liposomes, dendrimers, exosomes and gold nanoparticles, are designed to penetrate the blood‐brain barrier and deliver chemotherapy, radiotherapy, immunotherapy, gene therapy, phototherapy, magnetic hyperthermia and sonodynamic therapy for glioma.
Wenqian Jiang, Jing Fei, Rangrang Fan, Haifeng Chen, Liangxue Zhou, Gang Guo   +5 more
wiley   +1 more source

ALFA-1/C9orf72 regulates lipid metabolism through HLH-30/TFEB.

, 2020
(A) Representative images of Nile Red staining in N2, alfa-1(ok3062), HLH-30::GFP-expressing wild-type and HLH-30::GFP-expressing alfa-1(ok3062) mutant C. elegans after 1 day of starvation. Enlarged images of boxed areas are shown in each panel.
Yon Ju Ji (3359057), Tao Zhang (43681), Janet Ugolino (3359054), Jiou Wang (267956), Jiayin Lu (716730), Dohoon Kim (251220)   +5 more
core   +1 more source