Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

[Ketua Dobbs]

Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert

James Wilson, MD, PhD, recalls being struck by the devastating toll of rare diseases as a young physician in the 1980s. He set out on a path to correct the genes that cause these conditions, including spinal muscle atrophy (SMA), the most common inherited fatal disease in infants.
Source: Newly approved gene therapy for SMA is based on delivery vehicle discovered by Penn expert