IntroductionMyasthenia gravis (MG) is an autoimmune condition targeting the neuromuscular junction, which manifests with neuromuscular symptoms of varying severity and significant morbidity. The mainstay of treatment in MG is mitigation of the immune cascade with steroids and non-steroidal immunosuppressive therapies. The therapeutic strategies in MG are transitioning from broad and indiscriminate immunosuppression to novel agents targeting key steps in MG pathogenesis, including T cell activation, B cell proliferation, complement activation, maintenance of pathogenic antibody production, and proinflammatory cytokine production.Areas coveredIn this review, an overview of the pathogenesis of MG and traditional MG therapies is presented, followed by a discussion of the novel MG drugs that have been evaluated in phase 3 clinical trials with an emphasis on those which have received regulatory approval.Expert opinionNovel MG therapeutics belonging to the classes of complement inhibitors, neonatal Fc receptor (FcRn) inhibitors and B cell depletors, as well as the other emerging MG drugs in the pipeline constitute promising treatment strategies with potentially better efficacy and safety compared to the conventional MG treatments. However, further long-term research is needed in order to optimize the implementation of these new treatment options for the appropriate patient populations.
机构:
Hosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Inst Recerca St Pau, IR SantPau, Barcelona, Spain
Ctr Invest Biomed Red Enfermedades Raras CIBERER, Valencia, Spain
Univ Autonoma Barcelona, Dept Med, Barcelona, SpainHosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Reyes-Leiva, David
Carbayo, Alvaro
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Hosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Inst Recerca St Pau, IR SantPau, Barcelona, Spain
Ctr Invest Biomed Red Enfermedades Raras CIBERER, Valencia, Spain
Univ Autonoma Barcelona, Dept Med, Barcelona, SpainHosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Carbayo, Alvaro
Vesperinas-Castro, Ana
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Hosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Inst Recerca St Pau, IR SantPau, Barcelona, Spain
Ctr Invest Biomed Red Enfermedades Raras CIBERER, Valencia, Spain
Univ Autonoma Barcelona, Dept Med, Barcelona, SpainHosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Vesperinas-Castro, Ana
Rojas-Garcia, Ricard
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Hosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Inst Recerca St Pau, IR SantPau, Barcelona, Spain
Ctr Invest Biomed Red Enfermedades Raras CIBERER, Valencia, Spain
Univ Autonoma Barcelona, Dept Med, Barcelona, SpainHosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Rojas-Garcia, Ricard
Querol, Luis
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Hosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Inst Recerca St Pau, IR SantPau, Barcelona, Spain
Ctr Invest Biomed Red Enfermedades Raras CIBERER, Valencia, Spain
Univ Autonoma Barcelona, Dept Med, Barcelona, SpainHosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Querol, Luis
Turon-Sans, Janina
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Hosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Inst Recerca St Pau, IR SantPau, Barcelona, Spain
Ctr Invest Biomed Red Enfermedades Raras CIBERER, Valencia, Spain
Univ Autonoma Barcelona, Dept Med, Barcelona, SpainHosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Turon-Sans, Janina
Pla-Junca, Francesc
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Ctr Invest Biomed Red Enfermedades Raras CIBERER, Valencia, SpainHosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Pla-Junca, Francesc
Olive, Montse
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Hosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Inst Recerca St Pau, IR SantPau, Barcelona, Spain
Ctr Invest Biomed Red Enfermedades Raras CIBERER, Valencia, Spain
Univ Autonoma Barcelona, Dept Med, Barcelona, SpainHosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Olive, Montse
Gallardo, Eduard
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Hosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Inst Recerca St Pau, IR SantPau, Barcelona, Spain
Ctr Invest Biomed Red Enfermedades Raras CIBERER, Valencia, Spain
Univ Autonoma Barcelona, Dept Med, Barcelona, SpainHosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Gallardo, Eduard
Pujades-Rodriguez, Mar
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UCB Pharm, Brussels, BelgiumHosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Pujades-Rodriguez, Mar
Cortes-Vicente, Elena
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Hosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Inst Recerca St Pau, IR SantPau, Barcelona, Spain
Ctr Invest Biomed Red Enfermedades Raras CIBERER, Valencia, Spain
Univ Autonoma Barcelona, Dept Med, Barcelona, SpainHosp Santa Creu & Sant Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
机构:
Univ Toronto Neurol, Univ Hlth Network, Dept Med Neurol, Toronto, ON, CanadaUniv Toronto Neurol, Univ Hlth Network, Dept Med Neurol, Toronto, ON, Canada
Barnett, Carolina
Tabasinejad, Raha
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Univ Toronto Neurol, Univ Hlth Network, Dept Med Neurol, Toronto, ON, CanadaUniv Toronto Neurol, Univ Hlth Network, Dept Med Neurol, Toronto, ON, Canada
Tabasinejad, Raha
Bril, Vera
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Univ Toronto Neurol, Univ Hlth Network, Dept Med Neurol, Toronto, ON, CanadaUniv Toronto Neurol, Univ Hlth Network, Dept Med Neurol, Toronto, ON, Canada
机构:
Fdn IRCCS Ist Neurol Carlo Besta, Neuroimmunol & Neuromuscular Dis Unit, Milan, ItalyFdn IRCCS Ist Neurol Carlo Besta, Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Mantegazza, Renato
Antozzi, Carlo
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Fdn IRCCS Ist Neurol Carlo Besta, Neuroimmunol & Neuromuscular Dis Unit, Milan, ItalyFdn IRCCS Ist Neurol Carlo Besta, Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
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Univ Santiago De Compostela, Complexo Hosp Univ Santiago De Compostela, Fac Med, Serv Neurol,Unidad Enfermedades Neuromusculares, Santiago De Compostela, A Coruna, SpainComplexo Hosp Univ Ourense, Serv Neurol, Orense, Spain