Mutations that cause spinal muscular atrophy (SMA) may lead to abnormalities in the development of cells in the spinal cord, a study found.

Atamyo Therapeutics, a biotechnology company specializing in the development of next-generation gene therapies for limb-girdle muscular dystrophy (LGMD), announced at the MDA (Muscular Dystrophy ...

Partnership leverages AI-powered genome design to optimize AAV constructs, accelerate lead candidate selection, and improve drug substance manufacturability for rare and ultra-rare neuromuscular ...

This publication provides an overview of central nervous system (CNS) radiotracer development, discussing different aspects and stages of development. Non-invasive neuroimaging with radiotracers can ...