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
Lytras T, Kogevinas M, Kromhout H, Carsin AE, Anto JM, Bentouhami H, Weyler J, Heinrich J, Nowak D, Urrutia I, Martinez-Moratalla J, Gullon JA, Vega AP, Semjen CR, Pin I, Demoly P, Leynaert B, Villani S, Gislason T, Svanes O, Holm M, Forsberg B, Norback D, Mehta AJ, Probst-Hensch N, Benke G, Jogi R, Toren K, Sigsgaard T, Schlunssen V, Olivieri M, Blanc PD, Watkins J, Bono R, Buist AS, Vermeulen R, Jarvis D, Zock JP. Occupational exposures and incidence of chronic bronchitis and related symptoms over two decades: the European Community Respiratory Health Survey. Occup Environ Med. 2019 Apr;76(4):222-9. doi: 10.1136/oemed-2018-105274
Sunyer J, Jarvis D, Gotschi T, Garcia-Esteban R, Jacquemin B, Aguilera I, Ackerman U, de Marco R, Forsberg B, Gislason T, Heinrich J, Norback D, Villani S, Kunzli N. Chronic bronchitis and urban air pollution in an international study. Occup Environ Med. 2006 Dec;63(12):836-43. doi: 10.1136/oem.2006.027995