Listar Ciencias de la Salud por autor "Pintos-Morell, G."

Mostrando ítems 1-2 de 2

    • 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 ...

    • 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. ...