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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
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
Granero R, Penelo E, Stinchfield R, Fernandez-Aranda F, Aymami N, Gomez-Pena M, Fagundo AB, Sauchelli S , Islam MA, Menchon JM, Jimenez-Murcia S. Contribution of illegal acts to pathological gambling diagnosis: DSM-5 implications . J Addict Dis. 2014;33(1):41-52. doi: 10.1080/10550887.2014.882730
Kiladjian JJ, Gisslinger H, Passamonti F, Niederwieser D, Mendelson E, Sirulnik LA, Copley-Merriman K , Zhou X , Levy R, Knoops L, Cervantes F, Barbui T, Barosi G, Vannucchi A, Harrison C. Health-related quality of life (HRQoL) and symptom burden in patients (Pts) with myelofibrosis (MF) in the COMFORT-II study . Poster presented at the 17th Congress of the European Hematology Association; June 14, 2012. Amsterdam, the Netherlands. [abstract] Haematologica. 2012 Jul 16; 97(s1):154-5. Previously presented at the 2012 Annual Meeting of the American Society of Clinical Oncology (ASCO).
Nastaoupil LJ, Sinha R, Byrtek M, Zhou X , Taylor M, Friedberg J, Link B, Cerhan J, Dawson K, Flowers C. Characteristics and effectiveness of rituximab (R) maintenance regimens among US patients diagnosed with follicular lymphoma (FL) 2004-2007 . Poster presented at the 17th Congress of the European Hematology Association; June 14, 2012. Amsterdam, The Netherlands. [abstract] Haematologica. 2012 Jul 16; 97(s1):95-6. Previously presented at the 53rd Annual Meeting of the American Society of Hematology.