Documents
Treatment of adult MPSI mouse brains with IDUA-expressing mesenchymal stem cells decreases GAG deposition and improves exploratory behavior
Pereira Vanessa, Yasumura, Tenório Lígia, Lisboa, Matsumoto Priscila, Stilhano Roberta, Samoto, Calegare, Brandão Letícia, D’Almeida Vânia, Porcionatto Marimélia, Toma Leny, Nader Helena, Valero Valderez, Camassola Melissa, Nardi Nance, Han Sang, De, Ad, Filippo
Documents
Savoirs
Treatment of adult MPSI mouse brains with IDUA-expressing mesenchymal stem cells decreases GAG deposition and improves exploratory behavior
Pereira Vanessa, Yasumura, Tenório Lígia, Lisboa, Matsumoto Priscila, Stilhano Roberta, Samoto, Calegare, Brandão Letícia, D’Almeida Vânia, Porcionatto Marimélia, Toma Leny, Nader Helena, Valero Valderez, Camassola Melissa, Nardi Nance, Han Sang, De, Ad, Filippo
11 pages
English
Documents
A cross-sectional single-centre study on the spectrum of Pompe disease, German patients: molecular analysis of the GAA gene, manifestation and genotype-phenotype correlations
Herzog Andreas, Hartung Ralf, Reuser, Hermanns Pia, Runz Heiko, Karabul Nesrin, Gökce Seyfullah, Pohlenz Joachim, Kampmann Christoph, Lampe Christina, Beck Michael, Mengel Eugen
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Savoirs
A cross-sectional single-centre study on the spectrum of Pompe disease, German patients: molecular analysis of the GAA gene, manifestation and genotype-phenotype correlations
Herzog Andreas, Hartung Ralf, Reuser, Hermanns Pia, Runz Heiko, Karabul Nesrin, Gökce Seyfullah, Pohlenz Joachim, Kampmann Christoph, Lampe Christina, Beck Michael, Mengel Eugen
14 pages
English
Documents
Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy
Güngör Deniz, Reuser, Hagemans, De, Hop, Van
Documents
Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study
Hop, Karstens, Wokke, Kuks, Notermans, Faber, Verschuuren, Kruijshaar, Reuser, Van, De
Documents
Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies
Giugliani Roberto, Mehta Atul, Nicholls Kathy, Barisoni Laura, Jennette, Bragat Alexander, Castelli Jeff, Sitaraman Sheela, Lockhart, Germain, Hughes, Boudes
Documents
Savoirs
Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies
Giugliani Roberto, Mehta Atul, Nicholls Kathy, Barisoni Laura, Jennette, Bragat Alexander, Castelli Jeff, Sitaraman Sheela, Lockhart, Germain, Hughes, Boudes
11 pages
English
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