Advances in the understanding of disease mechanisms of autoimmune neuromuscular junction disorders.

Muscle weakness and fatigue are the hallmarks of autoimmune neuromuscular junction disorders. Although a plethora of immunosuppressive treatments exist, no cure is available to date and many patients are left with debilitating muscle weakness. Recent advances in the understanding of the structure and function of the neuromuscular junction, and the development of novel in vitro and in vivo models, have been instrumental in unravelling the pathophysiology of these autoimmune diseases. These advances are providing the rationale for the development of new therapeutic strategies. Restoration of the immune imbalance in these diseases, in parallel with symptomatic therapeutic approaches at the neuromuscular junction, will be crucial to obtain long-term remission or even cure.
Competing Interests: Declaration of interests MGH reports financial support paid to her institution from Leiden University Medical Center (OIO, 2017), Top Sector Life Sciences & Health to Samenwerkende Gezondheidsfondsen via the Target to B! consortium (LSHM18055-SGF), Prinses Beatrix Spierfonds (W.OR-17.13 and W.OR-19.13), and the Dutch Science Organization NWO (VENI 0915016181 0040); reports royalties for a diagnostic MuSK ELISA; and is a coinventor on two patent applications on MuSK-related research. AM is on the medical advisory board for the German Myasthenia Gravis Association. JJP reports research support paid to his institution from Association Française contre les Myopathies, Prinses Beatrix Spierfonds, the Dutch Science Organization NWO, and Alnylam Pharmaceuticals; and is a coinventor on two patent applications on MuSK-related research. RLP reports financial support paid to their institution from the Association Française contre les Myopathies. WDP reports financial support from the Lambert Initiative for Cannabinoid Therapeutics and Bridging Grants from the University of Sydney. The funders had no role in the writing of this paper.
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