Results 81 to 90 of about 854 (165)

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

Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)

, 2014
BackgroundMucopolysaccharidosis II, also known as Hunter syndrome, is a rare, X-linked disease caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase, which catalyses a step in the catabolism of glycosaminoglycans.
Silva, Laercio Antonio da [UNIFESP], Strufaldi, Maria Wany Louzada [UNIFESP], Maria Wany Louzada Strufaldi, Régis B Andriolo, Andriolo, Regis Bruni [UNIFESP], Laercio A Silva, Silva, Edina Mariko Koga da [UNIFESP], Edina MK da Silva   +7 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  

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, Jones, SA, Mendelsohn, NJ, Harmatz, P, FELICIANI, Claudio, Giugliani, R, S. Jones, Fang, J, Parini, R, Fang J, Harmatz P, FIUMARA, Agata, Parini R, HOS Natural History Working Group on behalf of HOS Investigators, Mendelsohn NJ, Jones SA, Giugliani R   +23 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

A biochemical and physicochemical comparison of two recombinant enzymes used for enzyme replacement therapies of hunter syndrome.

, 2014
Mucopolysaccharidosis II (MPS II, Hunter syndrome; OMIM 309900) is an X-linked lysosomal storage disease caused by a deficiency in the enzyme iduronate-2-sulfatase (IDS), leading to accumulation of glycosaminoglycans (GAGs).
Sohn, YB, Lee, J, Ko, AR, Sohn, JM, Jin, DK, Kwun, Y, Lee, JY, Yook, YJ, Chang, MS, Kim, CH, Chung, YK   +10 more
core   +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  

Comparisons of serum I2S pharmacokinetic parameters after 4 different idursulfase treatments.

, 2015
AUCinf, area under the concentration-time curve extrapolated to infinity; AUC0-t, area under the concentration-time curve from time 0 to the last sampling time; CL, total clearance; Cmax, maximum observed concentration; I2S, iduronate-2-sulfatase; IT-L ...
Hongsheng Xie (716868), Thomas G. McCauley (716871), Jou-Ku Chung (716869), Mary Ann Mascelli (716870)   +3 more
core   +1 more source

Review of the use of idursulfase in the treatment of mucopolysaccharidosis II.

Biologics : targets & therapy, 2011
Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is a heterogeneous, progressive X-linked recessively inherited lysosomal storage disease that is caused by a deficiency of the enzyme iduronate-2-sulfatase, resulting in abnormal tissue accumulation of the glycosaminoglycans, dermatan sulfate and heparan sulfate.
T Andrew, Burrow, Nancy D, Leslie
openaire   +1 more source