Navegação por Autor "Ramaswami, Uma"
Resultados 1-4 de 4
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Cardio-renal outcomes with long-term alfa enzyme replacement therapy : a 10-year Fabry outcome survey (FOS) analysis
Ramaswami, Uma; Beck, Michael; Hughes, Derralynn A.; Kampmann, Christoph; Botha, Jaco; Pintos-Morell, G.; West, Michael L.; Niu, Dauming; Nicholls, Kathy; Giugliani, Roberto (2019) [Artigo de periódico]Purpose: Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. Patients and methods: FOS (ClinicalTrials.gov ... -
A charitable access program for patients with lysosomal storage disorders in underserved communities worldwide
Mehta, Atul B.; Ramaswami, Uma; Muenzer, Joseph; Giugliani, Roberto; Ullrich, Kurt; Histed, Tanya Collin; Panahloo, Zoya; Wellhoefer, Hartmann; Frader, Joel (2021) [Artigo de periódico]Background: Lysosomal storage disorders (LSDs) are rare genetic disorders, with heterogeneous clinical manifesta‑ tions and severity. Treatment options, such as enzyme replacement therapy (ERT), substrate replacement ... -
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) [Artigo 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 ... -
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) [Artigo 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. ...