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Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I

Boy, Raquel; Schwartz, Ida Vanessa Doederlein; Krug, B??rbara C.; Silva, Luiz C. Santana da; Steiner, Carlos Eduardo; Acosta, Angelina Xavier; Ribeiro, Erlane M.; Galera, Marcial F.; Leivas, Paulo G. C.; Braz, Marlene (2011)

Long-term restoration of alpha-L-iduronidase activity in fibroblasts from patients with mucopolysaccharidosis type I after non-viral gene transfer

Poswar, Fabiano de Oliveira; Mayer, Fabiana Quoos; Burin, Maira Graeff; Matte, Ursula da Silveira; Giugliani, Roberto; Baldo, Guilherme (HCPA/FAMED/UFRGS, 2017)

Laronidase for treating mucopolysaccharidosis type I

El Dib, Regina Paolucci [UNIFESP]; Pastores, G. M. (Funpec-editora, 2007-01-01)

Mucopolysaccharidoses are a group of inherited metabolic diseases caused by the absence or deficiency of the lysosomal enzymes that are needed for breaking down glycosaminoglycans (GAGs). Over time, GAGs collect in cells, ...

Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I

Boy, Raquel; Schwartz, Ida Vanessa Doederlein; Krug, Bárbara C.; Silva, Luiz C. Santana da; Steiner, Carlos Eduardo; Acosta, Angelina Xavier; Ribeiro, Erlane M.; Galera, Marcial F.; Leivas, Paulo G. C.; Braz, Marlene; Boy, Raquel; Schwartz, Ida Vanessa Doederlein; Krug, Bárbara C.; Silva, Luiz C. Santana da; Steiner, Carlos Eduardo; Acosta, Angelina Xavier; Ribeiro, Erlane M.; Galera, Marcial F.; Leivas, Paulo G. C.; Braz, Marlene (2011)

Background/Aims Mucopolysaccharidosis type I (MPS I) is a rare lysosomal storage disorder treated with bone marrow transplantation or enzyme replacement therapy with laronidase, a high-cost orphan drug. Laronidase was ...

Mucopolysaccharidosis Type Ii: Identification Of 30 Novel Mutations Among Latin American Patients.

Brusius-Facchin, A C; Schwartz, I V D; Zimmer, C; Ribeiro, M G; Acosta, A X; Horovitz, D; Monlleó, I L; Fontes, M I B; Fett-Conte, A; Sobrinho, R P Oliveira; Duarte, A R; Boy, R; Mabe, P; Ascurra, M; de Michelena, M; Tylee, K L; Besley, G T N; Garreton, M C V; Giugliani, R; Leistner-Segal, S (2014)

Mutational and oxidative stress analysis in patients with mucopolysaccharidosis type I undergoing enzyme replacement therapy

Pereira, Vanessa Goncalves; Martins, Ana Maria; Micheletti, Cecilia; D'Almeida, Vania (Elsevier B.V., 2008-01-01)

Background: Mucopolysaccharidosis type I (NIPS 1) patients present a wide range of clinical manifestations, which could be due to the high molecular heterogeneity of the IDUA gene and to pathological events besides the ...

Identification and characterization of 13 new mutations in mucopolysaccharidosis type I patients

Matte, U; Yogalingam, G; Brooks, D; Leistner, S; Schwartz, I; Lima, L; Norato, DY; Brum, JM; Beesley, C; Winchester, B; Giugliani, R; Hopwood, JJ (Academic Press Inc Elsevier ScienceSan DiegoEUA, 2003)

Introduction to Brazilian Guidelines to Diagnosis, Treatment, and Monitoring for Gaucher Disease, Fabry Disease, Mucopolysaccharidosis I, and Pompe Disease

Martins, Ana Maria [UNIFESP] (Elsevier B.V., 2009-10-01)

Clinical and biochemical studies in mucopolysaccharidosis type II carriers

Schwartz, I. V. D.; Pinto, L. L. C.; Breda, G.; Lima, L.; Ribeiro, M. G.; Mota, J. G.; Acosta, Angelina Xavier; Correia, P.; Horovitz, Dafne Dain Gandelman; Porciuncula, C. G. G.; Figueiredo, E. Lipinski; Conte, A. C. Fett; Oliveira Sobrinho, R. P.; Norato, D. Y. J.; Paula, A. C.; Kim, C. A.; Duarte, A. R.; Boy, R.; Leistner-Segal, S.; Burin, M. G.; Giugliani, R. (Kluwer, 2014)

Analysis of male reproductive parameters in a murine model of mucopolysaccharidosis type I (MPS I)

Nascimento, Cinthia Castro do [UNIFESP]; Aguiar, Odair [UNIFESP]; D'Almeida, Vania [UNIFESP] (E-century Publishing Corp, 2014-01-01)

Mucopolysaccharidosis (MPS) I is a lysosomal storage disorder (LSD) that is characterised by alpha-L-idu-ronidase (Idua) deficiency and continuous deposition of glycosaminoglycans (GAGs), which consequently interferes with ...

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Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I

Long-term restoration of alpha-L-iduronidase activity in fibroblasts from patients with mucopolysaccharidosis type I after non-viral gene transfer

Laronidase for treating mucopolysaccharidosis type I

Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I

Mucopolysaccharidosis Type Ii: Identification Of 30 Novel Mutations Among Latin American Patients.

Mutational and oxidative stress analysis in patients with mucopolysaccharidosis type I undergoing enzyme replacement therapy

Identification and characterization of 13 new mutations in mucopolysaccharidosis type I patients

Introduction to Brazilian Guidelines to Diagnosis, Treatment, and Monitoring for Gaucher Disease, Fabry Disease, Mucopolysaccharidosis I, and Pompe Disease

Clinical and biochemical studies in mucopolysaccharidosis type II carriers

Analysis of male reproductive parameters in a murine model of mucopolysaccharidosis type I (MPS I)