Positive association between physical outcomes and patient-reported outcomes in late-onset Pompe disease: a cross sectional study

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Location: Deutsche Nationalbibliothek Frankfurt am Main

ISSN: 1750-1172

Extent: Online-Ressource

Language: Englisch

Notes: online resource.

Bibliographic citation: Positive association between physical outcomes and patient-reported outcomes in late-onset Pompe disease: a cross sectional study ; volume:15 ; number:1 ; day:3 ; month:9 ; year:2020 ; pages:1-8 ; date:12.2020
Orphanet journal of rare diseases ; 15, Heft 1 (3.9.2020), 1-8, 12.2020

Creator: Yuan, Meng
Andrinopoulou, Eleni-Rosalina
Kruijshaar, Michelle E.
Lika, Aglina
Harlaar, Laurike
Ploeg, Ans T. van der
Rizopoulos, Dimitris
Beek, Nadine A. M. E. van der

Contributor: SpringerLink (Online service)

DOI: 10.1186/s13023-020-01469-7

URN: urn:nbn:de:101:1-2020102419413468831149

Rights: Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.

Last update: 15.08.2025, 7:22 AM CEST

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Associated

Yuan, Meng
Andrinopoulou, Eleni-Rosalina
Kruijshaar, Michelle E.
Lika, Aglina
Harlaar, Laurike
Ploeg, Ans T. van der
Rizopoulos, Dimitris
Beek, Nadine A. M. E. van der
SpringerLink (Online service)

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Positive association between physical outcomes and patient-reported outcomes in late-onset Pompe disease: a cross sectional study

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Other Objects (12)

Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity

The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?

Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

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

Fatigue: an important feature of late-onset Pompe disease

Late-Onset Pompe Disease Presenting with Isolated Tongue Involvement

Forced vital capacity and cross-domain late-onset Pompe disease outcomes: an individual patient-level data meta-analysis

Walking delay as leading symptom in late-onset Pompe disease

Bright tongue sign in patients with late-onset Pompe disease

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

Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity

The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?

Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

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

Fatigue: an important feature of late-onset Pompe disease

Late-Onset Pompe Disease Presenting with Isolated Tongue Involvement

Forced vital capacity and cross-domain late-onset Pompe disease outcomes: an individual patient-level data meta-analysis

Walking delay as leading symptom in late-onset Pompe disease

Bright tongue sign in patients with late-onset Pompe disease

Alglucosidase alfa: 5 years of experience in late-onset Pompe disease

Analysis of voice quality in patients with late-onset Pompe disease

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Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity

The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?

Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

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

Fatigue: an important feature of late-onset Pompe disease

Late-Onset Pompe Disease Presenting with Isolated Tongue Involvement

Forced vital capacity and cross-domain late-onset Pompe disease outcomes: an individual patient-level data meta-analysis

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