Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study.
Howard JF Jr, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Muppidi S, Nowak RJ, O'Brien F, Wang JJ, Mantegazza R; REGAIN Study Group.
Lancet Neurol. 2017 Dec;16(12):976-986. doi: 10.1016/S1474-4422(17)30369-1. Epub 2017 Oct 20.
Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial.
Howard JF Jr, Bril V, Vu T, Karam C, Peric S, Margania T, Murai H, Bilinska M, Shakarishvili R, Smilowski M, Guglietta A, Ulrichts P, Vangeneugden T, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT Investigator Study Group.
Lancet Neurol. 2021 Jul;20(7):526-536. doi: 10.1016/S1474-4422(21)00159-9.
Myasthenia gravis.
Gilhus NE, Tzartos S, Evoli A, Palace J, Burns TM, Verschuuren JJGM.
Nat Rev Dis Primers. 2019 May 2;5(1):30. doi: 10.1038/s41572-019-0079-y.
Myasthenia gravis-Pathophysiology, diagnosis, and treatment.
Tannemaat MR, Huijbers MG, Verschuuren JJGM.
Handb Clin Neurol. 2024;200:283-305. doi: 10.1016/B978-0-12-823912-4.00026-8.
Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study.
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.
Lancet Neurol. 2023 May;22(5):383-394. doi: 10.1016/S1474-4422(23)00077-7.
Diagnosis and Management of Seronegative Myasthenia Gravis: Lights and Shadows.
Vinciguerra C, Bevilacqua L, Lupica A, Ginanneschi F, Piscosquito G, Rini N, Rossi A, Barone P, Brighina F, Di Stefano V.
Brain Sci. 2023 Sep 5;13(9):1286. doi: 10.3390/brainsci13091286.
Long-Term Efficacy and Safety of Ravulizumab in Adults With Anti-Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: Final Results From the Phase 3 CHAMPION MG Open-Label Extension.
Vu TH, Mantegazza R, Annane D, Katsuno M, Meisel A, Nicolle MW, Bril V, Aguzzi R, Frick G, Howard JF Jr; CHAMPION MG Study Group.
Eur J Neurol. 2025 Apr;32(4):e70158. doi: 10.1111/ene.70158.
Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis.
Howard JF Jr, Bril V, Burns TM, Mantegazza R, Bilinska M, Szczudlik A, Beydoun S, Garrido FJRR, Piehl F, Rottoli M, Van Damme P, Vu T, Evoli A, Freimer M, Mozaffar T, Ward ES, Dreier T, Ulrichts P, Verschueren K, Guglietta A, de Haard H, Leupin N, Verschuuren JJGM; Efgartigimod MG Study Group.
Neurology. 2019 Jun 4;92(23):e2661-e2673. doi: 10.1212/WNL.0000000000007600. Epub 2019 May 22.
A Phase 3 Trial of Inebilizumab in Generalized Myasthenia Gravis.
Nowak RJ, Benatar M, Ciafaloni E, Howard JF Jr, Leite MI, Utsugisawa K, Vissing J, Rojavin M, Li Q, Tang F, Wu Y, Rampal N, Cheng S; MINT Investigators.
N Engl J Med. 2025 Jun 19;392(23):2309-2320. doi: 10.1056/NEJMoa2501561. Epub 2025 Apr 8.
Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis.
Vu T, Meisel A, Mantegazza R, Annane D, Katsuno M, Aguzzi R, Enayetallah A, Beasley KN, Rampal N, Howard JF.
NEJM Evid. 2022 May;1(5):EVIDoa2100066. doi: 10.1056/EVIDoa2100066. Epub 2022 Apr 26.
