Results 71 to 80 of about 783 (129)

Surface plasmon resonance analysis of complex formation of therapeutic recombinant lysosomal enzymes with domain 9 of human cation-independent mannose 6-phosphate receptor

Molecular Genetics and Metabolism Reports, 2020
The efficacy of enzyme replacement therapy (ERT) for lysosomal storage diseases (LSDs) possibly depends on the cellular uptake of recombinant lysosomal enzymes (LEs), and it is known that cation-independent mannose 6-phosphate receptor (CI-M6PR) on the ...
Minori Kanzaki, Takahiro Tsukimura, Yasunori Chiba, Hitoshi Sakuraba, Tadayasu Togawa   +4 more
doaj   +1 more source

Eficácia e segurança da terapia com idursulfase em pacientes com mucopolissacaridose tipo II, com e sem comparação com placebo: revisão sistemática e metanálise

Cadernos de Saúde Pública, 2013
A mucopolissacaridose tipo II (MPS II) é uma doença genética de amplo espectro clínico, caracterizada por deficiência da enzima iduronato-2sulfatase.
Taciane Alegra, Dauana Pitano Eizerik, Caio César Silva de Cerqueira, Tiago V. Pereira, Alícia Dorneles Dornelles, Ida Vanessa Doederlein Schwartz   +5 more
doaj   +1 more source

Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey

, 2010
To use our experience with patients in the observational database HOS - the Hunter Outcome Survey - to evaluate the feasibility of home infusions of idursulfase for patients with mucopolysaccharidosis type II (MPS II)
Barbara K. Burton, Jones, SA, Burton, BK, GUFFON N, VAN DER PLOEG AT, Jane Roberts, van der Ploeg, Ans, SCARPA, MAURIZIO, BURTON BK, Guffon, N, Simon A. Jones, Feliciani, Claudio, ROBERTS J, Nathalie Guffon, Ans T. van der Ploeg, JONES SA, Roberts, J   +16 more
core   +1 more source

Successful desensitization to idursulfase in a patient with type II mucopolysaccharidosis (Hunter syndrome).

, 2011
Hunter syndrome or type II mucopolysaccharidosis is a rare lysosomal storage disease of X-linked recessive inheritance. It is characterized by a lack of the enzyme iduronate 2 sulfatase (I2S), which leads to the accumulation of glycosaminoglycans in many
Gomez, J F, Serrano Reyes, Carlos Daniel
core   +1 more source

A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4-7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy

, 2017
Purpose: The primary objective of this study was to determine the safety of idursulfase in Hunter syndrome patients aged 5 years or younger. ;Methods: Idursulfase (0.5 mg/kg) was administered intravenously on a weekly basis (52 infusions per patient) in ...
Giugliani, Roberto;Hwu, Wuh-Liang;Tylki-Szymanska, Anna;Whiteman, David A. H.;Pano, Arian, 胡務亮   +1 more
core   +1 more source

The effect of idursulfase on growth in patients with Hunter syndrome: data from the Hunter Outcome Survey (HOS)

, 2013
Hunter syndrome (mucopolysaccharidosis type II) is a rare and life-limiting multisystemic disorder with an X-linked recessive pattern of inheritance. Short stature is a prominent feature of this condition.
Jones, Simon A., Giugliani, Roberto, on behalf of the HOS Investigators, The HOS Natural History Working Group, Fang, Juanzhi, Harmatz, Paul, Mendelsohn, Nancy J., Gautschi, Matthias, Parini, Rossella   +7 more
core   +1 more source

Interim results of the first stage of a multicenter open multi-cohort study of the safety, pharmacokinetics, pharmacodynamics and efficacy of veranafusp alfa in adult patients with mucopolysaccharidosis type II

Фармация и фармакология (Пятигорск)
This article presents the interim results of the first stage (administration of the drug to patients aged ≥18 years with mucopolysaccharidosis type II) of a multicenter open multi-cohort phase II-III study (IDB-MPS-II-III), the aim of which was to assess
E. A. Lukina, R. V. Ponomarev, S. V. Trishina, E. S. Gabitova, N. D. Vashakmadze, G. A. Karkashadze, L. S. Namazova-Baranova   +6 more
doaj   +1 more source

Intracerebroventricular Enzyme Replacement Therapy in Patients with Neuropathic Form of Mucopolysaccharidosis Type II: to Help Practicing Physician

Вопросы современной педиатрии
Mucopolysaccharidosis type II (Hunter syndrome, MPS II) is a rare hereditary disease from the group of hereditary metabolic diseases. There are neuropathic and non-neuropathic forms of this disease. The neuropathic form is most common and leads to severe
Nataliya V. Zhurkova, Nato D. Vashakmadze, Georgiy A. Karkashadze, Elena V. Kaytukova   +3 more
doaj   +1 more source

Análise de impacto orçamentário da Idursulfase como terapia para o tratamento da Mucopolissacaridose tipo II na perspectiva do Sistema Único de Saúde [PDF]

, 2013
Com o crescente número da judicialização em saúde, o Estado tem cada vez mais comprometido seu orçamento para a aquisição de medicamentos sob ordem judicial.
Ramos, Maíra Catharina
core  

Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II

, 2008
Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare X-linked recessive disease caused by deficiency of the lysosomal enzyme iduronate-2-sulphatase.
Wraith, J. Edmond, Wraith, James
core   +1 more source