22 May 2018 - - New Albany, Ohio-based clinical stage rare disease gene therapy company Myonexus Therapeutics and Columbus, Ohio-based pediatric healthcare provider Nationwide Children's Hospital have been granted rare pediatric disease designation for the MYO-101 program, an AAV-based gene therapy for the treatment of limb girdle muscular dystrophy type 2E, by the US Food and Drug Administration, the company said.
Limb girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. In LGMD type 2E, disease usually occurs before age 10, progresses to loss of ambulation in the teen years, and often leads to death before age 30.
Myonexus Therapeutics is a developing treatments for limb girdle muscular dystrophies (LGMDs) based on research at Nationwide Children's Hospital.
The company's pipeline includes three clinical stage gene therapy programs (LGMD2E, LGMD2D, and LGMD2B) and two preclinical gene therapy programs (LGMD2C and LGMD2L).
Nationwide Children's Hospital is a not-for-profit freestanding pediatric healthcare system providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Children's has a staff of nearly 13,000 providing state-of-the-art pediatric care during more than 1.4 million patient visits annually.