Search Results - Simon Shohet

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  1. 1

    Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial by Alison Griffiths, Simon Shohet, Neil Johnson, Alasdair MacCulloch

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  2. 2

    Minimal clinically important differences in six-minute walking distance in late-onset Pompe disease by Kristl G. Claeys, Hani Kushlaf, Syed Raza, Noemi Hummel, Simon Shohet, Ian Keyzor, Agnieszka Kopiec, Ryan Graham, Brian Fox, Benedikt Schoser

    Published in Orphanet Journal of Rare Diseases (2024-04-01)
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  3. 3

    Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level... by Simon Shohet, Noemi Hummel, Shuai Fu, Ian Keyzor, Alasdair MacCulloch, Neil Johnson, Jeff Castelli, Ilona Czarny-Ozga, Tahseen Mozaffar, Howard Thom

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  4. 4

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

    Published in Journal of Patient-Reported Outcomes (2024-01-01)
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  5. 5

    Therapeutic Role of Pharmacological Chaperones in Lysosomal Storage Disorders: A Review of the Evidence and Informed Approach to Reclassification by Ian Keyzor, Simon Shohet, Jeff Castelli, Sheela Sitaraman, Biliana Veleva-Rotse, Jill M. Weimer, Brian Fox, Tobias Willer, Steve Tuske, Louise Crathorne, Klara J. Belzar

    Published in Biomolecules (2023-08-01)
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  6. 6

    A multi-country time and motion study to describe the experience and burden associated with the treatment of Fabry disease with enzyme replacement therapy with agalsidase alfa and... by Ian Keyzor, Ana Maria Martins, Sema Kalkan Uçar, Hiroyuki Yamakawa, Yin-Hsiu Chien, Nur Arslan, Dau-Ming Niu, Leyla Tümer, Laura Baldock, Simon Shohet, Joseph D. Giuliano

    Published in Orphanet Journal of Rare Diseases (2025-08-01)
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  7. 7

    P028: Switching treatment to cipaglucosidase alfa+miglustat positively affects motor function and quality of life in patients with late-onset Pompe disease by Priya Kishnani, Barry Byrne, Kristl Claeys, Paula Clemens, Jordi Díaz-Manera, Mazen Dimachkie, Hani Kushlaf, Tahseen Mozaffar, Mark Roberts, Antonio Toscano, Noemi Hummel, Fred Holdbrook, Simon Shohet, Benedikt Schoser

    Published in Genetics in Medicine Open (2024-01-01)
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  8. 8

    Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease by 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

    Published in Journal of Patient-Reported Outcomes (2024-11-01)
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