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It is estimated that one in 50,000 children have Myotubular Myopathy.

04.05.17 New step towards the treatment of myotubular myopathy

About AT132 for the treatment of X-linked Myotubular Myopathy

03.25.19 A Gene Therapy Tested in the Treatment of Myotubular Myopathy

Myotubular myopathy is a rare genetic disorder, affecting about one in 50,000 children.

Myotubular myopathy is a severe genetic disease that leads to muscle paralysis.

Myotubular myopathy is a rare genetic disorder, affecting about one in 50,000 children.

Efficacy of gene therapy demonstrated in canine and murine models of myotubular myopathy

This trial concerns myotubular myopathy, a rare and severe genetic disease affecting the skeletal muscles.

Efficacy of gene therapy demonstrated in canine and murine models of myotubular myopathy

Myotubular myopathy is caused by a lack of myotubularin, an enzyme that transforms the lipidic messengers.

Collaboration to develop gene therapy treatment in Myotubular Myopathy disease – Feb. 2014

This is particularly vital for rarer conditions, like myotubular and centronuclear myopathy.

Myotubular myopathy is caused by a lack of myotubularin, an enzyme that transforms the lipidic messengers.

My son died suddenly in 2005 of Viral Myocarditis.