A natural history study of X-linked myotubular myopathy

Conclusions:
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.

Source: Neurology - September 25, 2017 Category: Neurology Authors: Amburgey, K., Tsuchiya, E., de Chastonay, S., Glueck, M., Alverez, R., Nguyen, C.-T., Rutkowski, A., Hornyak, J., Beggs, A. H., Dowling, J. J. Tags: Muscle disease, Natural history studies (prognosis) ARTICLE Source Type: research