Listar Ciencias de la Salud por autor "Kampmann, Christoph"
Mostrando ítems 1-5 de 5
-
Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report
Giugliani, Roberto; Westwood, Stephanie; Wellhoefer, Hartmann; Schenk, Jörn Magnus; Gurevich, Andrey; Kampmann, Christoph (2018) [Artículo de periódico]Anderson-Fabry disease (AFD) is a rare lysosomal storage disorder. Randomized controlled clinical trials (RCTs) are preferred as the highest category of evidence, but limited availability of robust evidence in rare diseases ... -
Evaluation of the long-term treatment efects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling : data from the Hunter Outcome Survey (HOS)
Muenzer, Joseph; Botha, Jaco; Harmatz, Paul; Giugliani, Roberto; Kampmann, Christoph; Burton, Barbara K. (2021) [Artículo de periódico]Background: Mucopolysaccharidosis II (MPS II; Hunter syndrome) is a rare, life-limiting lysosomal storage disease caused by defcient iduronate-2-sulfatase activity. Enzyme replacement therapy (ERT) with intravenous (IV) ... -
Long-term outcomes with agalsidase alfa enzyme replacement therapy : analysis using deconstructed composite events
Beck, Michael; Hughes, Derralynn A.; Kampmann, Christoph; Pintos-Morell, G.; Ramaswami, Uma; West, Michael L.; Giugliani, Roberto (2017) [Artículo de periódico]This is a retrospective analysis of Fabry Outcome Survey data from children/adults (n =677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining cerebrovascular, cardiac, and renal morbidity ... -
The inflammation in the cytopathology of patients with mucopolysaccharidoses : immunomodulatory drugs as an approach to therapy
Wiesinger, Anna-Maria; Bigger, Brian; Giugliani, Roberto; Scarpa, Maurizio; Moser, Tobias; Lampe, Christina; Kampmann, Christoph; Lagler, Florian B. (2022) [Artículo de periódico]Mucopolysaccharidoses (MPS) are a group of lysosomal storage diseases (LSDs), characterized by the accumulation of glycosaminoglycans (GAGs). GAG storageinduced inflammatory processes are a driver of cytopathology in MPS ... -
Twenty years of the Fabry Outcome Survey (FOS) : insights, achievements, and lessons learned from a global patient registry
Beck, Michael; Ramaswami, Uma; Hernberg-Stahl, E.; Hughes, Derralynn A.; Kampmann, Christoph; Mehta, Atul B.; Nicholls, Kathleen M.; Niu, Dauming; Pintos-Morell, G.; Reisin, Ricardo; West, Michael L.; Schenk, Jörn Magnus; Anagnostopoulou, Christina; Botha, Jaco; Giugliani, Roberto (2022) [Artículo de periódico]Background: Patient registries provide long-term, real-world evidence that aids the understanding of the natural history and progression of disease, and the efects of treatment on large patient populations with rare diseases. ...