Detalles de la búsqueda
1.
Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial.
Mol Genet Metab
; 141(2): 108121, 2024 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-38184428
2.
Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study.
Mol Genet Metab
; 138(2): 106963, 2023 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-36481125
3.
Development of the Cold Agglutinin Disease Symptoms and Impact Questionnaire (CAD-SIQ).
Eur J Haematol
; 111(2): 211-219, 2023 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-37128840
4.
Clinical relevance of globotriaosylceramide accumulation in Fabry disease and the effect of agalsidase beta in affected tissues.
Mol Genet Metab
; 137(4): 328-341, 2022 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-36334424
5.
Patient-Reported Factors in Treatment Satisfaction in Patients with Relapsed/Refractory Multiple Myeloma (RRMM).
Oncologist
; 24(11): 1479-1487, 2019 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-31371520
6.
Clinical trials from the patient perspective: survey in an online patient community.
BMC Health Serv Res
; 17(1): 166, 2017 02 27.
Artículo
en Inglés
| MEDLINE | ID: mdl-28241758
7.
Patient-centered activity monitoring in the self-management of chronic health conditions.
BMC Med
; 13: 77, 2015 Apr 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-25889598
8.
A randomized controlled trial of an online self-management program for adults with arthritis pain.
J Behav Med
; 38(3): 483-96, 2015 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-25686538
9.
Changes in prevalence of prescription opioid abuse after introduction of an abuse-deterrent opioid formulation.
Pain Med
; 15(3): 440-51, 2014 Mar.
Artículo
en Inglés
| MEDLINE | ID: mdl-24330279
10.
Carotid intima-media thickness progression to predict cardiovascular events in the general population (the PROG-IMT collaborative project): a meta-analysis of individual participant data.
Lancet
; 379(9831): 2053-62, 2012 Jun 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-22541275
11.
Is the body adiposity index (hip circumference/height(1.5)) more strongly related to skinfold thicknesses and risk factor levels than is BMI? The Bogalusa Heart Study.
Br J Nutr
; 109(2): 338-45, 2013 Jan 28.
Artículo
en Inglés
| MEDLINE | ID: mdl-22716994
12.
Participatory research with an online drug forum: a survey of user characteristics, information sharing, and harm reduction views.
Subst Use Misuse
; 48(8): 661-70, 2013 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-23750771
13.
Measurement Properties of 2 Novel PROs, the Pompe Disease Symptom Scale and Pompe Disease Impact Scale, in the COMET Study.
Neurol Clin Pract
; 13(5): e200181, 2023 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-37559825
14.
Low birth weight is associated with higher blood pressure variability from childhood to young adulthood: the Bogalusa Heart Study.
Am J Epidemiol
; 176 Suppl 7: S99-105, 2012 Oct 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-23035149
15.
Differential humanistic and economic burden of mild, moderate and severe haemophilia in european adults: a regression analysis of the CHESS II study.
Orphanet J Rare Dis
; 17(1): 148, 2022 04 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-35379282
16.
Health-related quality of life, direct medical and societal costs among children with moderate or severe haemophilia in Europe: multivariable models of the CHESS-PAEDs study.
Orphanet J Rare Dis
; 17(1): 150, 2022 04 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-35379284
17.
Prevalence of atherosclerotic plaque in young and middle-aged asymptomatic individuals: the Bogalusa heart study.
South Med J
; 104(12): 803-8, 2011 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-22089358
18.
Cost-Effectiveness Analysis of rFVIIIFc Versus Contemporary rFVIII Treatments for Patients with Severe Hemophilia A Without Inhibitors in the United States.
Pharmacoecon Open
; 5(4): 625-633, 2021 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-34268704
19.
Development of the Fabry Disease Patient-Reported Outcome (FD-PRO): a new instrument to measure the symptoms and impacts of Fabry Disease.
Orphanet J Rare Dis
; 16(1): 285, 2021 06 25.
Artículo
en Inglés
| MEDLINE | ID: mdl-34172077
20.
Fabry Disease Patient-Reported Outcome (FD-PRO) demonstrates robust measurement properties for assessing symptom severity in Fabry disease.
Mol Genet Metab Rep
; 29: 100824, 2021 Dec.
Artículo
en Inglés
| MEDLINE | ID: mdl-34900595