العنوان البديل: Mecanismos inovadores de acesso pré-comercialização a tecnologias para doenças raras no Brasil: um estudo de caso de alfapabinafuspe para mucopolissacaridose tipo II. (Portuguese)

المؤلفون: Falavigna, Maicon, Borges Migliavaca, Celina, Tanaka, Elio, Santos, Marcos

المصدر: JBES: Brazilian Journal of Health Economics / Jornal Brasileiro de Economia da Saúde; Apr2023, Vol. 15 Issue 1, p67-70, 4p

مصطلحات موضوعية: RARE diseases, LYSOSOMAL storage diseases, MUCOPOLYSACCHARIDOSIS, ENZYME replacement therapy, ENZYME deficiency, PUBLIC goods

مصطلحات جغرافية: BRAZIL

المؤلفون: Reis, Matheus O., Cruz, Raquel A. S., Oliveira, Luiz G. S., Bassuino, Daniele M., Schwertz, Claiton I., Bianchi, Matheus V., Sonne, Luciana, Pavarini, Saulo P., Driemeier, David

المصدر: Journal of Veterinary Diagnostic Investigation; Jul2019, Vol. 31 Issue 4, p581-584, 4p

مصطلحات موضوعية: LYSOSOMAL storage diseases, COWS, EQUIDAE, AUTOPSY, DEER

مصطلحات جغرافية: BRAZIL, RIO Grande do Sul (Brazil : State)

المؤلفون: Costa-Motta, Fabiana Moura^1,2, Acosta, Angelina Xavier^3,4,5, Abé-Sandes, Kiyoko^4,6, Bender, Fernanda^1,2, Schwartz, Ida Vanessa D.^1,2,7, Giugliani, Roberto^1,2,3,7, Leistner-Segal, Sandra^1,2 ssegal@hcpa.ufrgs.br

المصدر: Molecular Genetics & Metabolism. Dec2011, Vol. 104 Issue 4, p603-607. 5p.

مصطلحات موضوعية: *MUCOPOLYSACCHARIDOSIS IV, *LYSOSOMAL storage diseases, *ARYLSULFATASES, *GENETIC mutation, *HAPLOTYPES

مصطلحات جغرافية: BRAZIL

المؤلفون: Müller, Karen B.¹, Rodrigues, Mayra D. B.¹, Pereira, Vanessa G.¹, Martins, Ana M.¹, D'Almeida, Vânia^1,2 vaniadalmeida@uol.com.br

المصدر: Diagnostic Pathology. 2010, Vol. 5, p65-68. 4p.

مصطلحات موضوعية: *LYSOSOMAL storage diseases, *INBORN errors of metabolism, *GLYCOGEN storage disease type II, *GENETIC disorders

مصطلحات جغرافية: BRAZIL

المؤلفون: Porsch, Daiana Benck¹ (AUTHOR), Nunes, Ane Cláudia Fernandes^2,3 (AUTHOR) nunes.acf@gmail.com, Milani, Vagner¹ (AUTHOR), Rossato, Liana Bertolin¹ (AUTHOR), Mattos, Cristiane Bastos¹ (AUTHOR), Tsao, Marilyn⁴ (AUTHOR), Netto, Cristina⁴ (AUTHOR), Burin, Maira⁴ (AUTHOR), Pereira, Fernanda⁴ (AUTHOR), Matte, Úrsula^4,5 (AUTHOR), Giugliani, Roberto^4,5,6 (AUTHOR), Barros, Elvino José Guardão^1,6,7 (AUTHOR)

المصدر: Renal Failure. 2008, Vol. 30 Issue 9, p825-830. 6p. 1 Black and White Photograph, 2 Diagrams, 1 Chart.

مصطلحات موضوعية: *LYSOSOMAL storage diseases, *HEMODIALYSIS, *INBORN errors of metabolism, *DIALYSIS (Chemistry)

مصطلحات جغرافية: BRAZIL

المؤلفون: Hastings, Caroline^1,2 (AUTHOR) caroline.hastings@ucsf.edu, Vieira, Camilo³ (AUTHOR) dr.camilovieira@gmail.com, Liu, Benny^4,5 (AUTHOR) beliu@alamedahealthsystem.org, Bascon, Cyrus¹ (AUTHOR) cyrus.bascon@ucsf.edu, Gao, Claire^6,7 (AUTHOR) Claire_gao@brown.edu, Wang, Raymond Y.^8,9 (AUTHOR) rawang@CHOC.ORG, Casey, Alicia¹⁰ (AUTHOR) Alicia.casey@childrens.harvard.edu, Hrynkow, Sharon¹¹ (AUTHOR) sharon.hrynkow@cyclodex.com

المصدر: Orphanet Journal of Rare Diseases. 10/21/2019, Vol. 14 Issue 1, pN.PAG-N.PAG. 1p.

مصطلحات موضوعية: *NIEMANN-Pick diseases, *LYSOSOMAL storage diseases, *ADULT-child relationships, *CASE studies, *INTRAVENOUS therapy, *SEQUESTRATION (Chemistry), *BUSULFAN, *YOUNG adults

مصطلحات جغرافية: BRAZIL

العنوان البديل: High cost drugs for rare diseases in Brazil: the case of lysosomal storage disorders. (English)

المؤلفون: Mônica Vinhas de Souza, Bárbara Corrêa Krug, Paulo Dornelles Picon, Ida Vanessa Doederlein Schwartz

المصدر: Revista Ciência & Saúde Coletiva; 2010, Vol. 15 Issue 7, p3443-3454, 12p, 3 Charts

مصطلحات موضوعية: LYSOSOMAL storage diseases, THERAPEUTICS, MEDICAL protocols, HEALTH policy, PUBLIC health

مصطلحات جغرافية: BRAZIL

العنوان البديل: High cost drugs for rare diseases in Brazil: the case of lysosomal storage disorders. (English)

المؤلفون: de Souza, Mônica Vinhas, Krug, Bárbara Corrêa, Picon, Paulo Dornelles, Schwartz, Ida Vanessa Doederlein

المصدر: Revista Ciência & Saúde Coletiva; 2010 supplement 3, Vol. 15, p3443-3454, 12p

مصطلحات موضوعية: LYSOSOMAL storage diseases, TREATMENT of rare diseases, DRUG prices, HEALTH policy, BRAZIL. Ministry of Health

مصطلحات جغرافية: BRAZIL

المؤلفون: Aranda, Carolina¹, Ensina, Luis Felipe¹, Solé, Dirceu¹, Martins, Ana Maria¹

المصدر: Molecular Genetics & Metabolism. Jan2017, Vol. 120 Issue 1/2, pS22-S23. 1p.

مصطلحات موضوعية: *LYSOSOMAL storage diseases, *THERAPEUTICS, *THERAPEUTIC use of enzymes, *ALLERGY desensitization, *MOLECULAR weights, *MEDICAL centers

مصطلحات جغرافية: BRAZIL

المؤلفون: Federhen, Andressa¹, de Cássia Cascaes Batista, Carla¹, Burin, Maira¹, Leistner-Segal, Sandra¹, Matte, Ursula¹, Rafaelli, Célio¹, Bender, Fernanda¹, Brusius, Ana Carolina¹, Pasqualim, Gabriela¹, Giugliani, Roberto¹

المصدر: Molecular Genetics & Metabolism. Feb2015, Vol. 114 Issue 2, pS43-S43. 1p.

مصطلحات موضوعية: *LYSOSOMAL storage diseases, *THERAPEUTICS, *DISEASE incidence, *EPIDEMIOLOGY, *COMPARATIVE studies, *DIAGNOSIS

مصطلحات جغرافية: BRAZIL