Publications

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Displaying 1 through 28 of 28 publications.

van der Pol M, Watson V, Boyers D. Stability of willingness to pay: does time and treatment allocation in a randomized controlled trial influence willingness to pay? Med Decis Making. 2024 Jul;44(5):470-80. doi: 10.1177/0272989X241249654.

Mansfield C, Boeri M, Coulter J, Baranowski E, Sparks S, An Haack K, Hamed A. The value of knowing: preferences for genetic testing to diagnose rare muscle diseases. Orphanet J Rare Dis. 2024 Apr 22;19(1):173. doi: 10.1186/s13023-024-03160-7

Yee KS, Alexanderian D, Martin S, Olayinka-Amao B, Whiteman DAH. Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment. Orphanet J Rare Dis. 2024 Apr 12;19(1):148. doi: 10.1186/s13023-024-03147-4

Yee KS, Lewis S, Evans E, Romano (DeMuro) C, Alexanderian D. Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II. Orphanet J Rare Dis. 2024 Mar 10;19(1):100. doi: 10.1186/s13023-024-03034-y

Yee KS, Chirila C, Davenport E, Mladsi D, Barnett C, Kronenberger WG. A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT. Orphanet J Rare Dis. 2023 Nov 2;18(1):343. doi: 10.1186/s13023-023-02957-2

Forsythe E, Haws RM, Argente J, Beales P, Martos-Moreno GA, Dollfus H, Chirila C, Gnanasakthy A, Buckley BC, Mallya UG, Clement K, Haqq AM. Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results. Orphanet J Rare Dis. 2023 Jan 16;18(1):12. doi: 10.1186/s13023-022-02602-4

Kuhnen P, Wabitsch M, von Schnurbein J, Chirila C, Mallya UG, Callahan P, Gnanasakthy A, Poitou C, Krabusch PM, Stewart M, Clement K. Quality of life outcomes in two phase 3 trials of setmelanotide in patients with obesity due to LEPR or POMC deficiency. Orphanet J Rare Dis. 2022 Feb 5;17(1):38. doi: 10.1186/s13023-022-02186-z

Foreman PK, Margulis AV, Alexander K, Shediac R, Calingaert B, Harding A, Pladevall-Vila M, Landis S. Birth prevalence of phenylalanine hydroxylase deficiency: a systematic literature review and meta-analysis. Orphanet J Rare Dis. 2021 Jun 3;16(1):253. doi: 10.1186/s13023-021-01874-6

Noel E, Dussol B, Lacombe D, Bedreddine N, Fouilhoux A, Ronco P, Genevaz D, Bekri S, Hagege A, Dupuis-Simeon F, Derrien Ansquer V, Germain DP, Lidove O. Treatment needs and expectations for Fabry disease in France: development of a new Patient Needs Questionnaire. Orphanet J Rare Dis. 2019 Dec 4;14(1):284. doi: 10.1186/s13023-019-1254-7

Vickers A. An evaluation of survival curve extrapolation techniques using long-term observational cancer data. Med Decis Making. 2019 Nov;39(8):926-38. doi: 10.1177/0272989X19875950

Watson V, Porteous T, Bolt T, Ryan M. Mode and frame matter: assessing the impact of survey mode and sample frame in choice experiments. Med Decis Making. 2019 Oct;39(7):827-41. doi: 10.1177/0272989X19871035

Paquin RS, Fischer R, Mansfield C, Mange B, Beaverson K, Ganot A, Martin AS, Morris C, Rensch C, Ricotti V, Russo LJ, Sadosky A, Smith EC, Peay H. Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients. Orphanet J Rare Dis. 2019 May 9;14(1):102. doi: 10.1186/s13023-019-1069-6

Harrington M, Whalley D, Twiss J, Rushton R, Martin S, Huynh L, Yang H. Insights into the natural history of metachromatic leukodystrophy from interviews with caregivers. Orphanet J Rare Dis. 2019 Apr 29;14(1):89. doi: 10.1186/s13023-019-1060-2

Cruden G, Spencer J, Dong OM. Improving the identification of adolescent depression through primary care screening approaches. Presented at the 40th Annual Meeting of the Society for Medical Decision Making; October 17, 2018. Montreal, Canada. [abstract] Med Decis Making. 2018 Dec 27; 39(1):E272. doi: 10.1177/0272989X18821191

Vass C, Rigby D, Tate K, Stewart A, Payne K. An exploratory application of eye-tracking methods in a discrete choice experiment. Med Decis Making. 2018 Aug;38(6):658-72. doi: 10.1177/0272989X18782197

Rowen D, Labeit A, Stevens K, Elliott J, Mulhern B, Carlton J, Basarir H, Brazier J. Estimating a preference-based single index measuring the quality-of-life impact of self-management for diabetes. Med Decis Making. 2018 Aug;38(6):699-707. doi: 10.1177/0272989X18784291

Wheeler AC, Sacco P, Cabo R. Unmet clinical needs and burden in Angelman syndrome: a review of the literature. Orphanet J Rare Dis. 2017 Oct 16;12(1):164. doi: 10.1186/s13023-017-0716-z

Barnett CL, Tomlins SA, Underwood DJ, Wei JT, Morgan TM, Montie JE, Denton BT. Two-stage biomarker protocols for early detection of prostate cancer. Med Decis Making. 2017 Oct;37(7):815-26. doi: 10.1177/0272989X17696996

Vass C, Rigby D, Payne K. The role of qualitative research methods in discrete choice experiments. Med Decis Making. 2017 Apr;37(3):298-313. doi: 10.1177/0272989X16683934

Peasgood T, Brennan A, Mansell P, Elliott J, Basarir H, Kruger J. The impact of diabetes-related complications on preference-based measures of health-related quality of life in adults with type I diabetes. Med Decis Making. 2016 Nov;36(8):1020-33. doi: 10.1177/0272989X16658660.

DiBenedetti DB, Brown TM, Danchenko N. Assessing patient and caregiver experiences with symptoms and behaviors associated with schizophrenia. J Depress Anxiety. 2016 Jul 17;5(3):240. doi: 10.4200/2167-1044.1000240

Brown TM, DiBenedetti DB, Danchenko N, Weiller E, Fava M. Symptoms of anxiety and irritability in patients with major depressive disorder. J Depress Anxiety. 2016 Jun 18;5:237. doi: 10.4200/2167-1044.1000237

Kruger J, Pollard D, Basarir H, Thokala P, Cooke D, Clark M, Bond R, Heller S, Brennan A. Incorporating psychological predictors of treatment response into health economic simulation models: a case study in type 1 diabetes. Med Decis Making. 2015 Oct;35(7):872-87. doi: 10.1177/0272989X15590143

Davis AE, Mehrotra S, Friedewald JJ, Daskin MS, Skaro AI, Abecassis MM, Ladner DP. Improving geographic equity in kidney transplantation using alternative kidney sharing and optimization modeling. Med Decis Making. 2015 Aug;35(6):797-807. doi: 10.1177/0272989X14557696

Bernfort L, Brodtkorb T. QALY som effektmått inom vården : möjligheter och begränsningar. Linköping: linköping University Electronic Press. Cmt- Rapport. 2012.

Brodtkorb T. National system for assessment, prioritization and introduction of non-pharmaceutical health technologies, a pilot study. Cmt- Rapport. 2010.

Eberth B, Watson V, Ryan M, Hughes J, Barnett G. Does one size fit all? Investigating heterogeneity in men's preferences for benign prostatic hyperplasia treatment using mixed logit analysis. Med Decis Making. 2009 Nov;29(6):707-15. doi: 10.1177/0272989X09341754

Earnshaw SR, Richter A, Sorensen SW, Hoerger TJ, Hicks KA, Engelgau M, Thompson T, Narayan KMV, Williamson DE, Gregg E, Zhang P. Optimal allocation of resources across four interventions for type 2 diabetes. Med Decis Making. 2002 Jan 1;22(5):S80-91.