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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
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.
Patel RI, Davis PJ, Orr RJ, Ferrari LR, Rimar S, Hannallah RS, Cohen IT, Colingo K, Donlon JV, Haberkern CM, McGowan FX, Prillaman BA , Parasuraman TV, Creed MR. Single-dose ondansetron prevents postoperative vomiting in pediatric outpatients . Anesth Analg. 1997 Sep;85(3):538-45. doi: 10.1097/00000539-199709000-00011