Publications
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Displaying 1 through 14 of 14 publications.
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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
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
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
Harries M, Mohr P, Grange F, Ehness R, Benjamin L, Siakpere O, Barth J, Stapelkamp C, Pfersch S, McLeod L, Wolowacz S, Kaye JA, Kontoudis I. Treatment patterns and outcomes of Stage IIIB/IIIC melanoma in France, Germany and the UK: a retrospective and prospective observational study (MELABIS). Int J Clin Pract. 2017 May 16;71(5):e12946. doi: 10.1111/ijcp.12946
Hirsch JD, Gnanasakthy A, Lale R, Choi K, Sarkin AJ. Efficacy of canakinumab vs. triamcinolone acetonide according to multiple gouty arthritis-related health outcomes measures. Int J Clin Pract. 2014 Dec;68(12):1503-7. doi: 10.1111/ijcp.12521
Davis AE, Mehrotra S, Friedewald J, Ladner D. Characteristics of a simulation model of the national kidney transplantation system. Poster presented at the 2013 Winter Simulation Conference: Simulation: Making Decisions in a Complex World; December 2013. [abstract] Ieee/Acm Trans Comput Biol Bioinform. 2013 Dec; p.2320-9.
Naslund MJ, Gilsenan AW, Midkiff KD, Bown A, Wolford ET, Wang A. Prevalence of lower urinary tract symptoms and prostate enlargement in the primary care setting. Int J Clin Pract. 2007 Sep 1;61(9):1437-45.
Berger A, Dukes E, Martin SA, Edelsberg J, Oster G. Characteristics and healthcare costs of patients with FMS. Int J Clin Pract. 2007 Sep 1;61(9):1498-508.