Simon Shohet
Marlow, ENG, United Kingdom
Last Updated: 09/26/2026
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Found 9 publications
A disease progression model comparing the long-term mobility and respiratory outcomes of adults with late-onset Pompe disease receiving cipaglucosidase alfa plus miglustat versus alglucosidase alfa.
Journal: Journal of comparative effectiveness research
Published: July 15, 2026
Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data.
Journal: Journal of comparative effectiveness research
Published: February 27, 2026
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 and aggregate data.
Journal: Journal of comparative effectiveness research
Published: September 17, 2024
Health-Related Quality-of-Life Utility Values in Adults With Late-Onset Pompe Disease: Analyses of EQ-5D Data From the PROPEL Clinical Trial.
Journal: Journal of health economics and outcomes research
Published: June 11, 2024
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease.
Journal: Journal of patient-reported outcomes
Published: April 05, 2024
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 agalsidase beta.
Journal: Orphanet journal of rare diseases
Published: February 28, 2024
Minimal clinically important differences in six-minute walking distance in late-onset Pompe disease.
Minimal clinically important differences in six-minute walking distance in late-onset Pompe disease.
Journal: Orphanet journal of rare diseases
Published: October 05, 2023
Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data.
Journal: Journal of patient-reported outcomes
Published: August 21, 2023
Therapeutic Role of Pharmacological Chaperones in Lysosomal Storage Disorders: A Review of the Evidence and Informed Approach to Reclassification.
Journal: Biomolecules
Published: June 20, 2023
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Last Updated: 09/26/2026