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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
Ribera A, Marsal JR, Faixedas MT, Rosas A, Tizon-Marcos H, Rojas S, Labata C, Cardenas M, Homs S, Tomas-Querol C, Garcia-Picart J, Roura G, Masotti M, Mauri J, Pijoan JI, Barrabes JA, Ferreira-Gonzalez I, Codi IAM Investigators Investigators. Revascularized ST-segment elevation myocardial infarction. Temporal trends in contemporary therapies and impact on outcomes. Rev Esp Cardiol. 2022 Aug;75(8):659-68. doi: 10.1016/j.rec.2021.10.011.
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
Harries M, Mohr P, Grange F, Ehness R, Benjamin L, Siakpere O, Barth J, Stapelkamp C, Pfersch S, McLeod L, Wolowacz S, Kaye JA, Kontoudis I. Treatment patterns and outcomes of Stage IIIB/IIIC melanoma in France, Germany and the UK: a retrospective and prospective observational study (MELABIS). Int J Clin Pract. 2017 May 16;71(5):e12946. doi: 10.1111/ijcp.12946
Ferreira-Gonzalez I, Permanyer Miralda G, Heras M, Ribera A, Marsal JR, Cascant P, Aros F, Bueno H, Sanchez PL, Cunat J, Civeira E, Marrugat J, Investigadores del Estudio MASCARA. Prognosis and management of patients with acute coronary syndrome and polyvascular disease. Rev Esp Cardiol. 2009 Sep;62(9):1012-21. doi: 10.1016/s1885-5857(09)73267-0
Ribera A, Ferreira-Gonzalez I, Cascant P, Marsal JR, Romero B, Pedrol D, Martinez-Useros C, Pons JM, Fernandez T, Permanyer-Miralda G, ARCA Study Investigators. Survival, clinical status and quality of life five years after coronary surgery. The ARCA study. Rev Esp Cardiol. 2009 Jun;62(6):642-51. doi: 10.1016/s1885-5857(09)72228-5