Selected Publications

Qarni TN, Jones FJS, Drachman B, Khella S, Pieretti J, Bustamante NS, Karam C.: Treatment characteristics of patients with hereditary transthyretin amyloidosis: a cohort study Orphanet J Rare Dis. : 2024 .

Traub R, Qarni T, Cohen AD, Karam C.: Paraproteinemic neuropathies Muscle Nerve. : 2024.

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. : 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. : 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 : 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. : 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.

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