Novel Drug Granted Orphan Designation for Rare Musculoskeletal Disorder

Audentes Therapeutics announced that AT001, an investigational drug in development for the treatment of X-Linked Myotubular Myopathy (XLMTM), has been granted Orphan Drug designation by the Food and Drug Administration (FDA).

XLMTM is a rare, severe, inherited disorder that affects skeletal muscles from birth. It is characterized by debilitating muscle weakness, respiratory impairment and early mortality, affecting approximately one in 50,000 newborn males worldwide. Currently, there is no available treatment for this condition.

RELATED: Musculoskeletal Disorders Resource Center

AT001 is a novel product based on adeno-associated virus (AAV) gene therapy technology that is designed to restore the skeletal muscle's ability to produce myotubularin.

For more information call (415) 638–6556 or visit AudentesTX.com.

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