Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis
Howard JF, Nowak RJ, Wolfe GI, Freimer ML, Vu TH, Hinton JL, Benatar M, Duda PW, MacDougall JE, Farzaneh-Far R, Kaminski HJ, Barohn R, Dimachkie M, Pasnoor M, Farmakidis C, Liu T, Colgan S, Benatar M, Bertorini T, Pillai R, Henegar R, Bromberg M, Gibson S, Janecki T, Freimer M, Elsheikh B, Matisak P, Genge A, Guidon A, David W, Habib A, Mathew V, Mozaffar T, Hinton J, Hewitt W, Barnett D, Sullivan P, Ho D, Howard J, Traub R, Chopra M, Kaminski H, Aly R, Bayat E, Abu-Rub M, Khan S, Lange D, Holzberg S, Khatri B, Lindman E, Olapo T, Sershon L, Lisak R, Bernitsas E, Jia K, Malik R, Lewis-Collins T, Nicolle M, Nowak R, Sharma A, Roy B, Nye J, Pulley M, Berger A, Shabbir Y, Sachdev A, Patterson K, Siddiqi Z, Sivak M, Bratton J, Small G, Kohli A, Fetter M, Vu T, Lam L, Harvey B, Wolfe G, Silvestri N, Patrick K, Zakalik K, Duda P, MacDougall J, Farzaneh-Far R, Pontius A, Hoarty M. Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis. JAMA Neurology 2020, 77: 582-592. PMID: 32065623, PMCID: PMC7042797, DOI: 10.1001/jamaneurol.2019.5125.Peer-Reviewed Original ResearchConceptsGeneralized myasthenia gravisSecondary end pointsDaily living scoreTolerability profileEnd pointLiving scoreMyasthenia gravisClinical effectsPlacebo-controlled phase 2 clinical trialKey secondary efficacy end pointsKey secondary end pointSecondary efficacy end pointsSevere Generalized Myasthenia GravisQuantitative Myasthenia Gravis ScorePhase 2 clinical trialAcetylcholine receptor autoantibodiesEfficacy end pointMyasthenia gravis scoreMG activityComplement component 5Disease-specific variablesQuality of lifeComplete complement inhibitionBroader populationDaily SCClinical characteristics of fibroblast growth factor receptor 3 antibody‐related polyneuropathy: a retrospective study
Kovvuru S, Cardenas Y, Huttner A, Nowak R, Roy B. Clinical characteristics of fibroblast growth factor receptor 3 antibody‐related polyneuropathy: a retrospective study. European Journal Of Neurology 2020, 27: 1310-1318. PMID: 32068339, DOI: 10.1111/ene.14180.Peer-Reviewed Original ResearchConceptsChronic inflammatory neuropathiesInflammatory neuropathiesSensory neuropathyFGFR3 antibodiesDistal lower extremity weaknessIntravenous immunoglobulin responseLower extremity paresthesiasCommon presenting symptomLower extremity weaknessRetrospective chart reviewPure sensory neuropathyHistory of cancerDistal sensationMotor findingsPresenting symptomChart reviewClinical characteristicsDemyelinating featuresNerve biopsySymptom onsetUnsteady gaitClinical presentationFoot dropRetrospective studyAutoimmune diseases