An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

We report on a preterm neonate of 30 weeks gestational age who presented with marked muscular hypotonia ... at birth and was diagnosed with congenital myotonic dystrophy. Neuroimaging at 36 ...

Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach ...

--(BUSINESS WIRE)--SonoThera™, a biotechnology company dedicated to treating the root cause of human diseases through developing the next generation of genetic medicines, today announced it will ...

LAMA2-RD, also known as merosin-deficient congenital muscular dystrophy type 1A (MDC1A), is a life-threatening genetic disease that causes progressive muscle weakness, loss of mobility ...