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physical function (pf) 20a  (ProMIS Neurosciences)

 
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    ProMIS Neurosciences physical function (pf) 20a
    Physical Function (Pf) 20a, supplied by ProMIS Neurosciences, used in various techniques. Bioz Stars score: 90/100, based on 1 PubMed citations. ZERO BIAS - scores, article reviews, protocol conditions and more
    https://www.bioz.com/result/physical function (pf) 20a/product/ProMIS Neurosciences
    Average 90 stars, based on 1 article reviews
    physical function (pf) 20a - by Bioz Stars, 2026-05
    90/100 stars

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    Patient-level responder analysis ( A ) and group-level analysis ( B ) of PROMIS physical and fatigue domains and R-PAct <t>outcomes</t> in ERT-experienced patients. Patients with a change from baseline >0 in PROMIS Physical Function or R-PAct, or <0 for PROMIS Fatigue, were classed as ‘responders.’ *For the Fatigue endpoint, a negative change from the baseline value indicated a better health outcome. alg alglucosidase alfa, CFBL change from baseline, CI confidence interval, cipa cipaglucosidase alfa, ERT enzyme replacement therapy, LS least squares, mig miglustat, pbo placebo, PROMIS Patient-Reported Outcome <t>Measurement</t> <t>Information</t> <t>System,</t> R-Pact Rasch-built Pompe-specific Activity
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    Patient-level responder analysis ( A ) and group-level analysis ( B ) of PROMIS physical and fatigue domains and R-PAct <t>outcomes</t> in ERT-experienced patients. Patients with a change from baseline >0 in PROMIS Physical Function or R-PAct, or <0 for PROMIS Fatigue, were classed as ‘responders.’ *For the Fatigue endpoint, a negative change from the baseline value indicated a better health outcome. alg alglucosidase alfa, CFBL change from baseline, CI confidence interval, cipa cipaglucosidase alfa, ERT enzyme replacement therapy, LS least squares, mig miglustat, pbo placebo, PROMIS Patient-Reported Outcome <t>Measurement</t> <t>Information</t> <t>System,</t> R-Pact Rasch-built Pompe-specific Activity
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    - physical function short form 20a  (ProMIS Neurosciences)
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    ProMIS Neurosciences - physical function short form 20a
    PROMs included in review
    Physical Function Short Form 20a, supplied by ProMIS Neurosciences, used in various techniques. Bioz Stars score: 90/100, based on 1 PubMed citations. ZERO BIAS - scores, article reviews, protocol conditions and more
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    Patient-level responder analysis ( A ) and group-level analysis ( B ) of PROMIS physical and fatigue domains and R-PAct outcomes in ERT-experienced patients. Patients with a change from baseline >0 in PROMIS Physical Function or R-PAct, or <0 for PROMIS Fatigue, were classed as ‘responders.’ *For the Fatigue endpoint, a negative change from the baseline value indicated a better health outcome. alg alglucosidase alfa, CFBL change from baseline, CI confidence interval, cipa cipaglucosidase alfa, ERT enzyme replacement therapy, LS least squares, mig miglustat, pbo placebo, PROMIS Patient-Reported Outcome Measurement Information System, R-Pact Rasch-built Pompe-specific Activity

    Journal: Journal of Patient-Reported Outcomes

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

    doi: 10.1186/s41687-024-00805-w

    Figure Lengend Snippet: Patient-level responder analysis ( A ) and group-level analysis ( B ) of PROMIS physical and fatigue domains and R-PAct outcomes in ERT-experienced patients. Patients with a change from baseline >0 in PROMIS Physical Function or R-PAct, or <0 for PROMIS Fatigue, were classed as ‘responders.’ *For the Fatigue endpoint, a negative change from the baseline value indicated a better health outcome. alg alglucosidase alfa, CFBL change from baseline, CI confidence interval, cipa cipaglucosidase alfa, ERT enzyme replacement therapy, LS least squares, mig miglustat, pbo placebo, PROMIS Patient-Reported Outcome Measurement Information System, R-Pact Rasch-built Pompe-specific Activity

    Article Snippet: PROs evaluated included the Subject’s Global Impression of Change (SGIC), Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short Form 20a, PROMIS Fatigue Short Form 8a, Rasch-built Pompe-specific Activity (R-PAct), and European Quality of Life-5 Dimensions 5 Response Levels (EQ-5D-5L).

    Techniques: Activity Assay

    PROMs included in review

    Journal: Orphanet Journal of Rare Diseases

    Article Title: Measuring health related quality of life (HRQoL) in Lysosomal Storage Disorders (LSDs): a rapid scoping review of available tools and domains

    doi: 10.1186/s13023-024-03256-0

    Figure Lengend Snippet: PROMs included in review

    Article Snippet: PROMIS - Physical Function Short Form 20a [ ] , , 1 , N , Physical function , N.

    Techniques: Activity Assay, Clinical Proteomics