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Aiyegbusi OL, Cruz Rivera S, Roydhouse J, Kamudoni P, Alder Y, Anderson N, Baldwin RM, Bhatnagar V, Black J, Bottomley A, Brundage M, Cella D, Collis P, Davies EH, Denniston AK, Efficace F, Gardner A, Gnanasakthy A, Golub RM, Hughes SE, Jeyes F, Kern S, King-Kallimanis BL, Martin A, McMullan C, Mercieca-Bebber R, Monteiro J, Peipert JD, Quijano-Campos JC, Quinten C, Rantell KR, Regnault A, Sasseville M, Schougaard LMV, Sherafat-Kazemzadeh R, Snyder C, Stover AM, Verdi R, Wilson R, Calvert MJ. Recommendations to address respondent burden associated with patient-reported outcome assessment. Nat Med. 2024 Mar;30(3):650-9. doi: 10.1038/s41591-024-02827-9
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
Auclair D, Mansfield C, Fiala MA, Chari A, Cole CE, Kaufman JL, Orloff GJ, Siegel DS, Zonder JA, Mange B, Yesil J, Dalal M, Mikhael JR. Preferences and priorities for relapsed multiple myeloma treatments among patients and caregivers in the United States. Patient Prefer Adherence. 2022 Mar 1;2022(16):573-85. doi: 10.2147/PPA.S345906
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
Aletaha D, Husni ME, Merola JF, Ranza R, Bertheussen H, Lippe R, Young P, Cappelleri JC, Brown TM, Ervin C, Hsu M-A, Fallon L. Treatment mode preferences in psoriatic arthritis: a qualitative multi-country study. Patient Prefer Adherence. 2020 Jun 8;2020(14):949-61. doi: 10.2147/PPA.S242336
Taylor PC, Betteridge N, Brown TM, Woolcott J, Kivitz AJ, Zerbini C, Whalley D, Olayinka-Amao O, Chen C, Dahl P, Ponce de Leon D, Gruben D, Fallon L. Treatment mode preferences in rheumatoid arthritis: moving toward shared decision-making. Patient Prefer Adherence. 2020 Jan 20;14:119-31. doi: 10.2147/PPA.S220714
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