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Publications
Duran CE, Riera-Arnau J, Abtahi S, Pajouheshnia R , Hoxhaj V, Gamba M, Alsina E, Martin-Perez M, Garcia-Posa P, Llorente-Garcia A, Gonzalez-Bermejo D, Ibanez L, Sabate M, Vidal X, Ballarin E, Sanfelix-Gimeno G, Rodriguez-Bernal C, Peiro S, Garcia-Sempere A, Sanchez-Saez F, Lentile V, Ingrasciotta Y, Guarneri C, Tanaglia M, Tari M, Herings R, Houben E, Swart-Polinder K, Holthuis E, Huerta C, Gini R, Roberto G, Bartolini C, Paoletti O, Limoncella G, Girardi A, Hyeraci G, Andersen M, Kristiansen SB, Hallgreen CE, Klungel O, Sturkenboom M. Impact of the 2018 revised Pregnancy Prevention Programme by the European Medicines Agency on the use of oral retinoids in females of childbearing age in Denmark, Italy, Netherlands, and Spain: an interrupted time series analysis . Front Pharmacol. 2023 Aug 17;14:1207976. doi: 10.3389/fphar.2023.1207976
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
Abdulkareem NM, Bhat R, Powell RT, Chikermane S, Yande S , Trinh L, Abdelnasser HY, Tabassum M, Ruiz A, Sobieski M, Nguyen ND, Johnson M, Kaipparettu BA, Johnson CA, Bond RA, Stephan C, Triveni MV. Screening of GPCR drugs for repurposing in breast cancer . Front Pharmacol. 2022 Dec 6;13:1049640. doi: 10.3389/fphar.2022.1049640
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