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Orphanet Journal of Rare Diseases
|
October 31, 2021
Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)
Joseph Muenzer, Jaco Botha, Paul Harmatz, et al.
Gene
|
October 11, 2012
Genotypic and phenotypic characterization of Brazilian patients with GM1 gangliosidosis
Fernanda Sperb, Filippo Vairo, Maira Burin, et al.
Molecular Genetics and Metabolism Reports
|
August 9, 2021
Impact of the COVID-19 pandemic on the standard of care for patients with lysosomal storage diseases: A survey of healthcare professionals in the Fabry, Gaucher, and Hunter Outcome Survey registries
Deborah Elstein, Roberto Giugliani, Joseph Muenzer, et al.
Cells, Tissues, Organs
|
July 23, 2011
Recombinant encapsulated cells overexpressing alpha-L-iduronidase correct enzyme deficiency in human mucopolysaccharidosis type I cells
Guilherme Baldo, Fabiana Quoos Mayer, Maira Burin, et al.
Ciencia & Saude Coletiva
|
February 20, 2010
[Is there an association between cancer mortality and agrotoxics use? A contribution to the debate]
Paulo Fernandes Costa Jobim, Luciana Neves Nunes, Roberto Giugliani, et al.
Arquivos Brasileiros De Oftalmologia
|
September 5, 2007
[Pseudo-glaucoma in type VI mucopolysaccharidosis: case report]
Diane Marinho, Ana Cecilia Mano Azevedo, Samuel Rymer, et al.
Orphanet Journal of Rare Diseases
|
May 6, 2025
Global variations in diagnostic methods and epidemiological estimates in Pompe disease: findings from a scoping review
Roberto Giugliani, Faryn Solomon, Hani Kushlaf, et al.
Pediatrics
|
February 5, 2008
Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome)
Rick Martin, Michael Beck, Christine Eng, et al.
Molecular Genetics and Metabolism
|
June 20, 2021
MPSBase: Comprehensive repository of differentially expressed genes for mucopolysaccharidoses
Luís Dias Ferreira Soares, Gerda Cristal Villalba Silva, Francyne Kubaski, et al.
Molecular Genetics and Metabolism
|
March 30, 2013
The effect of idursulfase on growth in patients with Hunter syndrome: data from the Hunter Outcome Survey (HOS)
Simon A Jones, Rossella Parini, Paul Harmatz, et al.
Page
of 49
Search research articles
Search
Showing results (71-80 of 482) with videos related to
Sort By:
Page
of 49
Orphanet Journal of Rare Diseases
|
October 31, 2021
Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)
Joseph Muenzer, Jaco Botha, Paul Harmatz, et al.
Gene
|
October 11, 2012
Genotypic and phenotypic characterization of Brazilian patients with GM1 gangliosidosis
Fernanda Sperb, Filippo Vairo, Maira Burin, et al.
Molecular Genetics and Metabolism Reports
|
August 9, 2021
Impact of the COVID-19 pandemic on the standard of care for patients with lysosomal storage diseases: A survey of healthcare professionals in the Fabry, Gaucher, and Hunter Outcome Survey registries
Deborah Elstein, Roberto Giugliani, Joseph Muenzer, et al.
Cells, Tissues, Organs
|
July 23, 2011
Recombinant encapsulated cells overexpressing alpha-L-iduronidase correct enzyme deficiency in human mucopolysaccharidosis type I cells
Guilherme Baldo, Fabiana Quoos Mayer, Maira Burin, et al.
Ciencia & Saude Coletiva
|
February 20, 2010
[Is there an association between cancer mortality and agrotoxics use? A contribution to the debate]
Paulo Fernandes Costa Jobim, Luciana Neves Nunes, Roberto Giugliani, et al.
Arquivos Brasileiros De Oftalmologia
|
September 5, 2007
[Pseudo-glaucoma in type VI mucopolysaccharidosis: case report]
Diane Marinho, Ana Cecilia Mano Azevedo, Samuel Rymer, et al.
Orphanet Journal of Rare Diseases
|
May 6, 2025
Global variations in diagnostic methods and epidemiological estimates in Pompe disease: findings from a scoping review
Roberto Giugliani, Faryn Solomon, Hani Kushlaf, et al.
Pediatrics
|
February 5, 2008
Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome)
Rick Martin, Michael Beck, Christine Eng, et al.
Molecular Genetics and Metabolism
|
June 20, 2021
MPSBase: Comprehensive repository of differentially expressed genes for mucopolysaccharidoses
Luís Dias Ferreira Soares, Gerda Cristal Villalba Silva, Francyne Kubaski, et al.
Molecular Genetics and Metabolism
|
March 30, 2013
The effect of idursulfase on growth in patients with Hunter syndrome: data from the Hunter Outcome Survey (HOS)
Simon A Jones, Rossella Parini, Paul Harmatz, et al.
Page
of 49