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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
Janssens R, Lang T, Vallejo A, Galinsky J, Plate A, Morgan K, Cabezudo E, Silvennoinen R, Coriu D, Badelita S, Irimia R, Anttonen M, Manninen RL, Schoefs E, Vandebroek M, Vanhellemont A, Delforge M, Stevens H, Simoens S, Huys I. Patient preferences for multiple myeloma treatments: a multinational qualitative study. Front Med (Lausanne). 2021 Jul 6;8:686165. doi: 10.3389/fmed.2021.686165
DiBenedetti D, Slota C, Wronski SL, Vradenburg G, Comer M, Callahan LF, Winfield J, Rubino I, Krasa HB, Hartry A, Wieberg D, Kremer IN, Lappin D, Martin AD, Frangiosa T, Biggar V, Hauber B. Assessing what matters most to patients with or at risk for Alzheimer's and care partners: a qualitative study evaluating symptoms, impacts, and outcomes. Alzheimers Res Ther. 2020 Jul 30;12(90). doi: 10.1186/s13195-020-00659-6
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
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
Vinceti M, Chiari A, Eichmuller M, Rothman KJ, Filippini T, Malagoli C, Weuve J, Tondelli M, Zamboni G, Nichelli PF, Michalke B. A selenium species in cerebrospinal fluid predicts conversion to Alzheimer's dementia in persons with mild cognitive impairment. Alzheimers Res Ther. 2017 Dec 19;9(1):100. doi: 10.1186/s13195-017-0323-1
Bertelsen RJ, Rava M, Carsin AE, Accordini S, Benediktsdottir B, Dratva J, Franklin KA, Heinrich J, Holm M, Janson C, Johannessen A, Jarvis DL, Jogi R, Leynaert B, Norback D, Omenaas ER, Raherison C, Sanchez-Ramos JL, Schlunssen V, Sigsgaard T, Dharmage SC, Svanes C. Clinical markers of asthma and IgE assessed in parents before conception predict asthma and hayfever in the offspring. Clin Exp Allergy. 2017 May;47(5):627-38. doi: 10.1111/cea.12906