CONTEXT

Myasthenia gravis (MG) is a neuromuscular disorder in which functional acetylcholine receptors (AChR) become depleted at neuromuscular junctions due to an antibody-mediated autoimmune attack on the neuromuscular synapse. AChR antibodies can be detected in the serum of ~90% of patients with generalised MG. It is a rare orphan disease with a prevalence of 1.6–2 per 10,000 people and an incidence of 1.2–1.5 per 100,000 people in the EU.

Current treatments include symptomatic pharmacotherapy, immunosuppressive medication, immunomodulating therapies and a surgical procedure (thymectomy). Symptomatic pharmacotherapy using acetylcholinesterase inhibitors increases the availability of acetylcholine (ACh) at the neuromuscular junctions. Immunosuppressive medications include prednisone, cyclosporine, azathioprine, mycophenolate mofetil and occasionally cyclophosphamide. Immunomodulating approaches include plasmapheresis and the use of intravenous immunoglobulin (IVIg).

MYASTERIX project is expected to bring a more targeted therapeutic approach with fewer and less severe side effect than the existing therapeutics.