Myonexus Therapeutics is a clinical stage gene therapy company developing first ever corrective treatments for Limb-girdle muscular dystrophies (LGMD), rare progressive, monogenic diseases initially characterized by wasting and weakness of the voluntary muscles of the hip and shoulder areas that often eventually spreads to other muscles, including the heart.  In aggressive subtypes, symptoms begin between 3-4 years of age and continually progress with most patients becoming wheel chair dependent in their teens and mortality beginning by age 30.  No therapies currently exist.
Myonexus’ pipeline is licensed from Nationwide Children’s Hospital (Columbus, Ohio, USA), a global leader in muscular dystrophy gene therapy translational R&D, and includes three clinical stage assets and two preclinical stage assets:  LGMD2D (Phase 1 IM study completed with Phase 1/2a isolated limb perfusion study ongoing), LGMD2B (Phase 1 IM study ongoing), LGMD2E (systemic IV Phase 1/2a scheduled to begin November 2017), LGMD2L (preclinical), and LGMD2C (preclinical).
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