Selected Publications

Singer M, Khella S, Bird S, McIntosh P, Paudyal B, Wadhwani A, Quinn C, Karam C.: Single institution experience with efgartigimod in patients with myasthenia gravis: Patient selection, dosing schedules, treatment response, and adverse events Muscle Nerve. : 2023.

Bril V, Howard JF Jr, Karam C, De Bleecker JL, Murai H, Utsugisawa K, Ulrichts P, Brauer E, Zhao S, Mantegazza R, Vu T; ADAPT Study Group.: Effect of efgartigimod on muscle group subdomains in participants with generalized myasthenia gravis: post hoc analyses of the phase 3 pivotal ADAPT study Eur J Neurol. : 2023.

Morganroth J, Ljungberg L, Goldstein A, Kallish S, Asher SB, Quinn C, Price RS, Karam C.: Adult-Onset Thymidine Kinase Gene (TK2) Deficiency Neurology. : 2023.

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.

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