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

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Location: Deutsche Nationalbibliothek Frankfurt am Main

ISSN: 1750-1172

Extent: Online-Ressource

Language: Englisch

Notes: online resource.

Bibliographic citation: Qualitative interviews to improve patient-reported outcome measures in late-onset Pompe disease: the patient perspective ; volume:16 ; number:1 ; day:12 ; month:10 ; year:2021 ; pages:1-12 ; date:12.2021
Orphanet journal of rare diseases ; 16, Heft 1 (12.10.2021), 1-12, 12.2021

Creator: Hamed, Alaa
An Haack, Kristina
Gwaltney, Chad
Baranowski, Eileen
Stewart, Andrew
Krupnick, Robert
Tyler, Margaret
Sparks, Susan
Paty, Jean

Contributor: SpringerLink (Online service)

DOI: 10.1186/s13023-021-02067-x

URN: urn:nbn:de:101:1-2021122620014133588793

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

Last update: 15.08.2025, 7:31 AM CEST

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Hamed, Alaa
An Haack, Kristina
Gwaltney, Chad
Baranowski, Eileen
Stewart, Andrew
Krupnick, Robert
Tyler, Margaret
Sparks, Susan
Paty, Jean
SpringerLink (Online service)

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

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Other Objects (12)

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

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

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Late-Onset Pompe Disease Presenting with Isolated Tongue Involvement

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

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Walking delay as leading symptom in 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

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

Fatigue: an important feature of late-onset Pompe disease

Late-Onset Pompe Disease Presenting with Isolated Tongue Involvement

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

Bright tongue sign in patients with late-onset Pompe disease

Walking delay as leading symptom in late-onset Pompe disease

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

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

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

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

Fatigue: an important feature of late-onset Pompe disease

Late-Onset Pompe Disease Presenting with Isolated Tongue Involvement

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

Bright tongue sign in patients with late-onset Pompe disease

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