The UNC technology, developed by Pediatrics Professor Chengwen Li, M.D., Ph.D., uses adeno-associated viruses (AAVs) to ferry ...

Greg Friberg - Executive Vice President, Chief Research and Development Officer Good day ... later this year from both BMN 351 for Duchenne Muscular Dystrophy and BMN 333, our long-acting CNP ...

The FDA has placed another clinical hold on a gene therapy for Duchenne muscular dystrophy developed ... microdystrophin – to counteract the deficient production of the protein in muscles.

Evox development platform is concentrated ... at using exosomes to deliver dystrophin, a protein deficient in people with Duchenne muscular dystrophy. Last year, it also won a £1.5 million ...

London, UK-based Vicebio raised $100 million in a Series B that will help to fund the development of its Molecular ... leading to muscular weakness, seizures, developmental delays, and organ ...

Getsios, D., Blume, S., Ishak, K. J., Maclaine, G., & Hernández, L. (2012). An economic evaluation of early assessment for Alzheimer's disease in the ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.

Using a new fluorescent mouse model with advanced imaging techniques, researchers have successfully visualized how musculoskeletal components are integrated into the functional locomotor system during ...