Skip to main content
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
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
Strober B, Sigurgeirsson B, Popp G, Sinclair R, Krell J, Stonkus S, Septe M, Elewski BE, Gottlieb AB, Zhao Y, Tran MH, Karpov A, McLeod LD, Mordin M, Papavassils C, Nyirady J, Lebwohl M. Secukinumab improves patient-reported psoriasis symptoms of itching, pain, and scaling: results of two phase 3, randomized, placebo-controlled clinical trials. Int J Dermatol. 2016 Apr;55(4):401-7. doi: 10.1111/ijd.13236
Strober B, Sigurgeirsson B, Popp G, Sinclair R, Krell J, Stonkus S, Septe M, Elewski BE, Gottlieb AB, Zhao Y, Tran MH, Karpov A, McLeod LD, Mordin M, Papavassilis C, Nyirady J, Lebwohl M. Secukinumab improves patient‐reported psoriasis symptoms of itching, pain, and scaling: results of two phase 3, randomized, placebo‐controlled clinical trials. Int J Dermatol. 2016 Apr;55(4):401-7. doi: 10.1111/ijd.13236
Strober B, Zhao Y, Tran MH, Gnanasakthy A, Nyirady J, Papavassils C, Nelson LM, McLeod LD, Mordin M, Gottlieb A, Elewski B, Lebwohl M. Psychometric validation of the psoriasis symptom diary using phase III study data from patients with chronic plaque psoriasis. Int J Dermatol. 2016 Mar;55(3):e147-55. doi: 10.1111/ijd.13117