Skip to main content
Publications
Hauber B, Paulsen R, Krasa HB, Vradenburg G, Comer M, Callahan LF, Winfield J, Potashman M, Hartry A, Lee D, Wilson H, Hoffman DL, Weiberg D, Kremer IN, Taylor GA, Taylor JM, Lappin D, Martin AD, Frangiosa T, Biggar V, Slota C, Romano (DeMuro) C, DiBenedetti D. Assessing what matters to people affected by Alzheimer's disease: a quantitative analysis. Neurol Ther. 2023 Apr;12(2):505-27. doi: 10.1007/s40120-023-00445-0
DiBenedetti D, Menne H, Paulsen R, Krasa HB, Vradenburg G, Comer M, Callahan LF, Winfield J, Potashman M, Heithoff K, Hartry A, Oberdhan D, Wilson H, Hoffman DL, Weinberg D, Kremer IN, Taylor GA, Taylor JM, Lappin D, Martin AD, Hauber B, Romano (DeMuro) C. Technical review of clinical outcomes assessments across the continuum of Alzheimer's disease. Neurol Ther. 2023 Apr;12(2):571-95. doi: 10.1007/s40120-023-00443-2
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
Forns J, Dudukina E, Hagg D, Szentkuti P, Gembert K, Plana E, Gilsenan A, Horvath-Puho E, Ehrenstein V, Reutfors J, Rebordosa C. Risk of major cardiovascular and cerebrovascular events in users of lisdexamfetamine and other medications for attention-deficit/hyperactivity disorder in Denmark and Sweden: a population-based cohort study. Neurol Ther. 2022 Dec 1;11(4):1659-76. doi: 10.1007/s40120-022-00396-y
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