Detalhe da pesquisa
1.
The definition of neuronopathic Gaucher disease.
J Inherit Metab Dis
; 43(5): 1056-1059, 2020 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32242941
2.
The Digital 1-Minute Walk Test: A New Patient-centered Cardiorespiratory Endpoint.
Am J Respir Crit Care Med
; 209(6): 753-756, 2024 Mar 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-38206760
3.
Accuracy of serum procalcitonin for the diagnosis of sepsis in neonates and children with systemic inflammatory syndrome: a meta-analysis.
BMC Infect Dis
; 17(1): 302, 2017 04 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-28438138
4.
Video-based assessments of activities of daily living: generating real-world evidence in pediatric rare diseases.
Expert Rev Pharmacoecon Outcomes Res
; : 1-9, 2024 Jun 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-38789406
5.
A patient-centred and multi-stakeholder co-designed observational prospective study protocol: Example of the adolescent experience of treatment for X-linked hypophosphataemia (XLH).
PLoS One
; 19(1): e0295080, 2024.
Artigo
em Inglês
| MEDLINE | ID: mdl-38241270
6.
Protocol for a mixed-methods study to develop and feasibility test a digital system for the capture of patient-reported outcomes (PROs) in patients receiving chimeric antigen receptor T-cell (CAR-T) therapies (the PRO-CAR-T study).
BMJ Open
; 14(3): e085392, 2024 Mar 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-38553074
7.
Recommendations to address respondent burden associated with patient-reported outcome assessment.
Nat Med
; 30(3): 650-659, 2024 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-38424214
8.
So You Want to Build Your Disease's First Online Patient Registry: An Educational Guide for Patient Organizations Based on US and European Experience.
Patient
; 16(3): 183-199, 2023 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-36947286
9.
Accelerometer-derived sleep measures in idiopathic dystonia: A UK Biobank cohort study.
Brain Behav
; 13(9): e2933, 2023 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-37547976
10.
A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative.
Orphanet J Rare Dis
; 18(1): 195, 2023 07 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-37480076
11.
Use of remote monitoring and integrated platform for the evaluation of sleep quality in adult-onset idiopathic cervical dystonia.
J Neurol
; 270(3): 1759-1769, 2023 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-36414751
12.
Patient-reported, health economic and psychosocial outcomes in patients with Friedreich ataxia (PROFA): protocol of an observational study using momentary data assessments via mobile health app.
BMJ Open
; 13(8): e075736, 2023 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-37527887
13.
Co-production of a feasibility trial of pacing interventions for Long COVID.
Res Involv Engagem
; 9(1): 18, 2023 Mar 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-36997975
14.
Towards the international interoperability of clinical research networks for rare diseases: recommendations from the IRDiRC Task Force.
Orphanet J Rare Dis
; 18(1): 109, 2023 05 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-37161573
15.
Considerations for patient and public involvement and engagement in health research.
Nat Med
; 29(8): 1922-1929, 2023 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-37474660
16.
Development and usability testing of an electronic patient-reported outcome (ePRO) solution for patients with inflammatory diseases in an Advanced Therapy Medicinal Product (ATMP) basket trial.
J Patient Rep Outcomes
; 7(1): 98, 2023 10 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-37812323
17.
Precision Medicine for More Oxygen (P4O2)-Study Design and First Results of the Long COVID-19 Extension.
J Pers Med
; 13(7)2023 Jun 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-37511673
18.
Time to See the Difference: Video Capture for Patient-Centered Clinical Trials.
Patient
; 15(4): 389-397, 2022 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-34993934
19.
Patient-led development of digital endpoints and the use of computer vision analysis in assessment of motor function in rare diseases.
Front Pharmacol
; 13: 916714, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-36172196
20.
Feasibility of a new electronic patient-reported outcome (ePRO) system for an advanced therapy clinical trial in immune-mediated inflammatory disease (PROmics): protocol for a qualitative feasibility study.
BMJ Open
; 12(9): e063199, 2022 09 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36691123