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Journal of Inherited Metabolic Disease
|
November 10, 2012
Chronic kidney disease in adolescent and adult patients with phenylketonuria
Julia B Hennermann, Sylvia Roloff, Jutta Gellermann, et al.
Drug Design, Development and Therapy
|
June 26, 2020
Analysis of Renal and Cardiac Outcomes in Male Participants in the Fabry Outcome Survey Starting Agalsidase Alfa Enzyme Replacement Therapy Before and After 18 Years of Age
Rossella Parini, Guillem Pintos-Morell, Julia B Hennermann, et al.
Molecular Genetics and Metabolism
|
September 13, 2023
Screening for health-related quality of life and its determinants in Fabry disease: A cross-sectional multicenter study
Yaroslav Winter, Max Hilz, Felix Beuschlein, et al.
Molecular Genetics and Metabolism
|
May 3, 2018
Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis
Paul Harmatz, Federica Cattaneo, Diego Ardigò, et al.
Orphanet Journal of Rare Diseases
|
May 4, 2019
Growth charts for patients with Sanfilippo syndrome (Mucopolysaccharidosis type III)
Nicole M Muschol, Daniel Pape, Kai Kossow, et al.
Journal of Inherited Metabolic Disease
|
February 28, 2023
Long-term safety and efficacy of velmanase alfa treatment in children under 6 years of age with alpha-mannosidosis: A phase 2, open label, multicenter study
Nathalie Guffon, Vassiliki Konstantopoulou, Julia B Hennermann, et al.
JIMD Reports
|
July 13, 2022
Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation
Stefanie Maier, Miroslav Zivicnjak, Lorenz Grigull, et al.
Klinische Padiatrie
|
November 28, 2018
Retrospective Analysis of Whole-Body Magnetic Resonance Imaging of Bone Manifestations in Long-Term Treated Patients with Gaucher Disease Type 1
André Lollert, Katharina Laudemann, Eugen Mengel, et al.
Journal of Inherited Metabolic Disease
|
October 23, 2024
Natural history of valve disease in patients with mucopolysaccharidosis II and the impact of enzyme replacement therapy
Christoph Kampmann, Christina Lampe, Christiane M Wiethoff, et al.
The Journal of Pediatrics
|
November 26, 2019
The Clinical and Molecular Spectrum of GM1 Gangliosidosis
Laila Arash-Kaps, Katalin Komlosi, Marlene Seegräber, et al.
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Search research articles
Search
Showing results (31-40 of 97) with videos related to
Sort By:
Page
of 10
Journal of Inherited Metabolic Disease
|
November 10, 2012
Chronic kidney disease in adolescent and adult patients with phenylketonuria
Julia B Hennermann, Sylvia Roloff, Jutta Gellermann, et al.
Drug Design, Development and Therapy
|
June 26, 2020
Analysis of Renal and Cardiac Outcomes in Male Participants in the Fabry Outcome Survey Starting Agalsidase Alfa Enzyme Replacement Therapy Before and After 18 Years of Age
Rossella Parini, Guillem Pintos-Morell, Julia B Hennermann, et al.
Molecular Genetics and Metabolism
|
September 13, 2023
Screening for health-related quality of life and its determinants in Fabry disease: A cross-sectional multicenter study
Yaroslav Winter, Max Hilz, Felix Beuschlein, et al.
Molecular Genetics and Metabolism
|
May 3, 2018
Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis
Paul Harmatz, Federica Cattaneo, Diego Ardigò, et al.
Orphanet Journal of Rare Diseases
|
May 4, 2019
Growth charts for patients with Sanfilippo syndrome (Mucopolysaccharidosis type III)
Nicole M Muschol, Daniel Pape, Kai Kossow, et al.
Journal of Inherited Metabolic Disease
|
February 28, 2023
Long-term safety and efficacy of velmanase alfa treatment in children under 6 years of age with alpha-mannosidosis: A phase 2, open label, multicenter study
Nathalie Guffon, Vassiliki Konstantopoulou, Julia B Hennermann, et al.
JIMD Reports
|
July 13, 2022
Predictors of growth patterns in children with mucopolysaccharidosis I after haematopoietic stem cell transplantation
Stefanie Maier, Miroslav Zivicnjak, Lorenz Grigull, et al.
Klinische Padiatrie
|
November 28, 2018
Retrospective Analysis of Whole-Body Magnetic Resonance Imaging of Bone Manifestations in Long-Term Treated Patients with Gaucher Disease Type 1
André Lollert, Katharina Laudemann, Eugen Mengel, et al.
Journal of Inherited Metabolic Disease
|
October 23, 2024
Natural history of valve disease in patients with mucopolysaccharidosis II and the impact of enzyme replacement therapy
Christoph Kampmann, Christina Lampe, Christiane M Wiethoff, et al.
The Journal of Pediatrics
|
November 26, 2019
The Clinical and Molecular Spectrum of GM1 Gangliosidosis
Laila Arash-Kaps, Katalin Komlosi, Marlene Seegräber, et al.
Page
of 10