Selected Publications

Berends M, Nienhuis HLA, Adams D, Karam C, Luigetti M, Polydefkis M, Reilly MM, Sekijima Y, Hazenberg BPC. : Neurofilament Light Chains in Systemic Amyloidosis: A Systematic Review Int J Mol Sci. Mar 2024.

Howard JF Jr, Bril V, Vu T, Karam C, Peric S, De Bleecker JL, Murai H, Meisel A, Beydoun SR, Pasnoor M, Guglietta A, Van Hoorick B, Steeland S, T'joen C, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT+ Study Group.: Long-term safety, tolerability, and efficacy of efgartigimod (ADAPT+): interim results from a phase 3 open-label extension study in participants with generalized myasthenia gravis. Front. Neurol. January 2024.

Karam C, Mauermann ML, Gonzalez-Duarte A, Kaku MC, Ajroud-Driss S, Brannagan TH 3rd, Polydefkis M. : Diagnosis and treatment of hereditary transthyretin amyloidosis with polyneuropathy in the United States: Recommendations from a panel of experts. Muscle Nerve January 2024.

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. Nov 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. Oct 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. August 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 May 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. April 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 April 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 April 2023.