Measurement Properties of 2 Novel PROs, the Pompe Disease Symptom Scale and Pompe Disease Impact Scale, in the COMET Study-Reference-Cited by-同舟云学术

Measurement Properties of 2 Novel PROs, the Pompe Disease Symptom Scale and Pompe Disease Impact Scale, in the COMET Study

Published:2023-10 Issue:5 Volume:13 Page:
ISSN:2163-0402
Container-title:Neurology Clinical Practice
language:en
Short-container-title:Neur Clin Pract

Author:

Dimachkie Mazen M.¹^ORCID,Kishnani Priya S.¹,Ivanescu Cristina¹,Flore Giulio¹,Gwaltney Chad¹,van der Beek Nadine A.M.E.¹^ORCID,Hamed Alaa¹,An Haack Kristina¹,Pollissard Laurence¹,Baranowski Eileen¹,Sparks Susan E.¹,DasMahapatra Pronabesh¹,

Affiliation:

1. University of Kansas Medical Center (MMD), Kansas City, KS; Duke University Medical Center (PSK), Durham, NC; IQVIA Netherland (CI, GF), Amsterdam-Zuidoost, North Holland Province, The Netherlands; Gwaltney Consulting (CG), Westerly, RI; Center for Lysosomal and Metabolic Diseases (NAMEB), Department of Neurology, Erasmus MC University Medical Center, Rotterdam, The Netherlands; Sanofi (AH, EB, SES, PD), Cambridge, MA; and Sanofi (KAH, LP), Chilly-Mazarin, France.

Publisher

Ovid Technologies (Wolters Kluwer Health)

Subject

Neurology (clinical)

Link

https://www.neurology.org/doi/pdfdirect/10.1212/CPJ.0000000000200181

Reference21 articles.

1. Reuser A, Hirschhorn R, Kroos M. Part 16: Lysosomal disorders: Pompe disease: glycogen storage disease Type II, acid α-glucosidase (acid Maltase) deficiency. In: Valle D, Antonarakis S, Ballabio A, Beaudet A, Mitchell G, eds. OMMBID – The Online Metabolic and Molecular Bases of Inherited Diseases. McGraw-Hill Education, 2019.

2. Pompe's disease

3. Qualitative interviews to improve patient-reported outcome measures in late-onset Pompe disease: the patient perspective

4. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

5. Individual-patient monitoring in clinical practice: are available health status surveys adequate?

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