Dwight Koeberl

Dwight Koeberl is a PhD researcher at Duke University Medical Center | DUMC · Division of Medical Genetics, specializing in medical genetics and gene therapy.

 

Treatment With A New Drug Strengthens The Muscles Of Patients With Pompe Disease

Pompe disease is a rare and potentially fatal metabolic myopathy (prevalence 1 in 20,000). In Pompe disease, the deficiency of lysosomal acid α-glucosidase (GAA) leads to accumulation of lysosomal glycogen and muscle damage. Enzyme replacement therapy (ERT) for Pompe disease […]