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Publications
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
Ali MS, Berencsi K, Marinier K, Deltour N, Perez-Gutthann S, Pedersen L, Rijnbeek P, Lapi F, Simonetti M, Reyes C, Van der Lei J, Sturkenboom M, Prieto-Alhambra D. Comparative cardiovascular safety of strontium ranelate and bisphosphonates: a multi-database study in 5 EU countries by the EU-ADR Alliance. Osteoporos Int. 2020 Dec;31(12):2425-38.
Berencsi K, Sami A, Ali MS, Marinier K, Deltour N, Perez-Gutthann S, Pedersen L, Rijnbeek P, Van der Lei J, Lapi F, Simonetti M, Reyes C, Sturkenboom MCJM, Prieto-Alhambra D. Impact of risk minimisation measures on the use of strontium ranelate in Europe: a multi-national cohort study in 5 EU countries by the EU-ADR Alliance. Osteoporos Int. 2020 Apr;31(4):721-55. doi: 10.1007/s00198-020-05314-2
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
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
See CW, Srinivasan M, Saravanan S, Oldenburg CE, Esterberg EJ, Ray KJ, Glaser TS, Tu EY, Zegans ME, McLeod SD, Acharya NR, Lietman TM. Prior elicitation and Bayesian analysis of the Steroids for Corneal Ulcers Trial. Ophthalmic Epidemiol. 2012 Dec;19(6):407-13.
Gutierrez L, Roskell N, Rycroft C, Castellsague J, Beard S, Abeysinghe S, Shannon P, Robbins S, Gitlin M. Economic and clinical burden of non-vertebral non-hip (Nvnh), vertebral and multiple fractures in postmenopausal women in the United Kingdom. Poster presented at IOF World Congress on Osteoporosis & 10th European Congress on Clinical and Economic Aspects of Osteoporosis and Osteoarthritis; May 2010. Florence, Italy. [abstract] Osteoporos Int. 2010 May 1; 21(Suppl. 1):S96.