Specific binding of collagen Q to the neuromuscular junction is exploited to cure congenital myasthenia and to explore bases of myasthenia gravis

► The C-terminal domain of collagen Q binds to MuSK and perlecan. ► Congenital defects of collagen Q cause endplate acetylcholinesterase deficiency. ► Protein-anchoring therapy for collagen Q is proposed and proven in Colq−/− mice. ► MuSK autoantibody accounts for 5-15% of myasthenia gravis. ► MuSK-IgG blocks binding of MuSK to collagen Q.