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The Myosana platform might represent a potentially safer alternative to AVV to deliver the MTM1 gene to muscle.
X-Linked-Myotubular-Myopathy X-linked myotubular myopathy (MTM) is a neuromuscular disorder caused by mutations in the myotubularin (MTM1) gene.
It is a rare condition (estimated at 1:50,000 male births) associated with substantial morbidities and early mortality.
muscle-cs-2 MTM is a childhood disease characterized by profound, neonatal onset weakness, severe disabilities (including wheelchair and ventilator dependence), and early death.
At present, there are no treatments or disease-modifying therapies.
PDB_1m7r_EBI Previous attempts to treat MTM with AAV gene therapy tragically led to the death of a number of patients enrolled in the clinical trial.
Early indications of those patients who did not suffer serious adverse events in the trial are that delivery of the MTM1 gene to muscle has led to substantial improvements in muscle function.
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