Selected Publications
Bril V, Drużdż A, Grosskreutz J, Habib AA, Mantegazza R, Sacconi S, Utsugisawa K, Vissing J, Vu T, Boehnlein M, Bozorg A, Gayfieva M, Greve B, Woltering F, Kaminski HJ; MG0003 study team.: Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study Lancet Neurol 2023; 22: 383–94 : 2023.
Kishnani PS, Diaz-Manera J, Toscano A, Clemens PR, Ladha S, Berger KI, Kushlaf H, Straub V, Carvalho G, Mozaffar T, Roberts M, Attarian S, Chien YH, Choi YC, Day JW, Erdem-Ozdamar S, Illarioshkin S, Goker-Alpan O, Kostera-Pruszczyk A, van der Ploeg AT, An Haack K, Huynh-Ba O, Tammireddy S, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group.: Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial JAMA Neurol. : 2023.
Nicolas Sarmiento Bustamante, Taha Qarni, Felipe Jorge Simoes Jones, Sami Khella1, Brian Drachman, Janice Pieretti, Chafic Karam: Characteristics of a single-center cohort of patients with hereditary amyloidosis AAN abstract : 2023.
Taha Qarni, Nicolas Sarmiento Bustamante, Felipe Jorge Simoes Jones, Sami Khella, Brian Drachman, Janice Pieretti, Chafic Karam: Treatment characteristics in a cohort of patients with hereditary transthyretin amyloidosis AAN abstract : 2023.
Chafic Karam, Julian Gillmore, Gengshi Chen, Nia C Jenkins, Michael J Hale, Gemma MJ Taylor, Jersey Chen, Nicholas Viney, Eugene Schneider: A Multi-Method Approach to an Indirect Treatment Comparison of Eplontersen and Vutrisiran for the Treatment of Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy
AAN abstract : 2023.
Michael Polydefkis; Emre Aldinc; Nienhuis, ; Karam, Chafic ; Senda Ajroud-Driss; 関島良樹 ; Márcia Waddington Cruz ; Jennifer Barnes ; Paul Nioi ; Simina Ticau: NfL Levels Significantly Decrease in Response to Treatment with Patisiran or Vutrisiran in hATTR Amyloidosis with Polyneuropathy AAN abstract : 2023.
Mamatha Pasnoor, Vera Bril, Chafic Karam, Stojan Peric, Jan L. De Bleecker, Hiroyuki Murai, Andreas Meisel, Said Beydoun, Tuan Vu, Peter Ulrichts, Benjamin Van Hoorick, 10 Caroline T’joen, 10 Kimiaki Utsugisawa, 11 Jan Verschuuren, Renato Mantegazza, James F. Howard Jr, in collaboration with the ADAPT Investigator Study Group: Longterm Safety, Tolerability, and Efficacy of Efgartigimod
in Patients With Generalized Myasthenia Gravis:
Concluding Analyses From the ADAPT+ Study
AAN abstract : 2023.
Yarlas A, Lovley A, Brown D, Vera-Llonch M, Khella S, Karam C.: The impact of inotersen on Neuropathy Impairment Score in patients with hereditary transthyretin amyloidosis with polyneuropathy. BMC Neurol. : 108,2023.
Kimiaki Utsugisawa, 1 James F. Howard, Jr, 2 Vera Bril, 3, 4 Tuan Vu, 5 Chafic Karam, 6 Stojan Peric, 7 Jan L. De Bleecker, 8 Hiroyuki Murai, 9 Mamatha Pasnoor, 10 Francesco Sacca, 11 Andreas Meisel, 12 Antonio Guglietta, 13 Caroline T’joen, 13 Renato Mantegazza, 14 in collaboration with the ADAPT Investigator Study Group: Efgartigimod Treatment of Anti-Acetylcholine Receptor Antibody Negative Myasthenia Gravis Patients Japanese Society for Neuroimmunology (JSNI) 2022 : 2022.
Aggarwal R, Charles-Schoeman C, Schessl J, Bata-Csörgő Z, Dimachkie MM, Griger Z, Moiseev S, Oddis C, Schiopu E, Vencovský J, Beckmann I, Clodi E, Bugrova O, Dankó K, Ernste F, Goyal NA, Heuer M, Hudson M, Hussain YM, Karam C, Magnolo N, Nelson R, Pozur N, Prystupa L, Sárdy M, Valenzuela G, van der Kooi AJ, Vu T, Worm M, Levine T; ProDERM Trial Group.: Trial of Intravenous Immune Globulin in Dermatomyositis. N Engl J Med.;387(14):1264-1278. 387 (14): 1264-1278,2022.
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