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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)

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

Boy, R; Schwartz, IVD; Krug, BC; Santana-da-Silva, LC; Steiner, CE; Acosta, AX; Ribeiro, EM; Galera, MF; Leivas, PGC; Braz, M (B M J Publishing GroupLondonInglaterra, 2011)

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 ...

Conflitos bioéticos decorrentes do acesso a medicamentos órfãos no Brasil: o exemplo da laronidase para o tratamento da mucopolissacaridose tipo IBioethical conflicts arising out of access to orphan drugs in Brazil: the example of laronidase for treating mucopolysaccharidosis type I

Silva, Raquel Tavares Boy da (2018)

Conflitos bioéticos decorrentes do acesso a medicamentos órfãos no Brasil: o exemplo da laronidase para o tratamento da mucopolissacaridose tipo I

Silva, Raquel Tavares Boy da (2011)

Esta pesquisa busca, como seu objetivo principal, discutir conflitos morais decorrentes do acesso e da alocação de recursos públicos, a medicamentos órfãos, tomando como exemplo a laronidase, medicamento pleiteado para o ...

Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series

Horovitz, Dafne Dain Gandelman; Acosta, Angelina X.; Giugliani, Roberto; Hlavata, Anna; Hlavata, Katarina; Tchan, Michel C.; Barth, Anneliese Lopes; Cardoso Jr., Laercio; Leao, Emilia Katiane Embirucu de Araujo; Esposito, Ana Carolina; Kyosen, Sandra Obikawa [UNIFESP]; Souza, Carolina Fischinger Moura de; Martins, Ana Maria [UNIFESP] (Biomed Central Ltd, 2016)

Background: Enzyme replacement therapy (ERT) with laronidase (recombinant human alpha-L-iduronidase, Aldurazyme (R)) is indicated for non-neurological signs and symptoms of mucopolysaccharidosis type I (MPS I). The approved ...

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

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

Laronidase for treating mucopolysaccharidosis type I

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

Conflitos bioéticos decorrentes do acesso a medicamentos órfãos no Brasil: o exemplo da laronidase para o tratamento da mucopolissacaridose tipo IBioethical conflicts arising out of access to orphan drugs in Brazil: the example of laronidase for treating mucopolysaccharidosis type I

Conflitos bioéticos decorrentes do acesso a medicamentos órfãos no Brasil: o exemplo da laronidase para o tratamento da mucopolissacaridose tipo I

Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series

Laronidase hypersensitivity and desensitization of mucopolysaccharidosis I (Scheie syndrome) patient

Laronidase hypersensitivity and desensitization of mucopolysaccharidosis I (Scheie syndrome) patient

Laronidase hypersensitivity and desensitization in type I mucopolysaccharidosis: a case report

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

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

Laronidase for treating mucopolysaccharidosis type I

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

Conflitos bioéticos decorrentes do acesso a medicamentos órfãos no Brasil: o exemplo da laronidase para o tratamento da mucopolissacaridose tipo IBioethical conflicts arising out of access to orphan drugs in Brazil: the example of laronidase for treating mucopolysaccharidosis type I

Conflitos bioéticos decorrentes do acesso a medicamentos órfãos no Brasil: o exemplo da laronidase para o tratamento da mucopolissacaridose tipo I

Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series

Laronidase hypersensitivity and desensitization of mucopolysaccharidosis I (Scheie syndrome) patient

Laronidase hypersensitivity and desensitization of mucopolysaccharidosis I (Scheie syndrome) patient

Laronidase hypersensitivity and desensitization in type I mucopolysaccharidosis: a case report