Arama Sonuçları - Priya S. Kishnani

Characterization of a canine model of glycogen storage disease type IIIa Yazar: Haiqing Yi, Beth L. Thurberg, Sarah Curtis, Stephanie Austin, John Fyfe, Dwight D. Koeberl, Priya S. Kishnani, Baodong Sun

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Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity Yazar: Melodi Harfouche, Priya S. Kishnani, Eva Krusinska, Jamie Gault, Sheela Sitaraman, Amanda Sowinski, Irina Katz, Stephanie Austin, Margi Goldstein, Andrew E. Mulberg

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Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data Yazar: Priya S. Kishnani, Simon Shohet, Syed Raza, Noemi Hummel, Jeffrey P. Castelli, Sheela Sitaraman Das, Heng Jiang, Agnieszka Kopiec, Ian Keyzor, Andreas Hahn

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Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease Yazar: Priya S. Kishnani, Barry J. Byrne, Kristl G. Claeys, Jordi Díaz-Manera, Mazen M. Dimachkie, Hani Kushlaf, Tahseen Mozaffar, Mark Roberts, Benedikt Schoser, Noemi Hummel, Agnieszka Kopiec, Fred Holdbrook, Simon Shohet, Antonio Toscano, on behalf of the PROPEL Study Group

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