The natural history of X-linked myotubular myopathy shows a more stable disease course than previously predicted, with improved survival through infancy and early childhood.
MTM is one of the most severe neuromuscular disorders, with affected individuals requiring extensive mechanical interventions for survival. However, among study participants, the disease course was more stable than predicted, with more individuals surviving infancy and early childhood. These data reflect the disease burden of MTM but offer hope in terms of future therapeutic intervention.
Amburgey et al. (Sat,) studied this question.