Motor neuron disease research breakthrough
Researchers have found that the insulin-like growth factor 1 (IGF-1) dramatically increases the in vitro growth of corticospinal motor neuron (CSMN) axons – projections that carry nerve impulses
Ultragenyx Pharmaceutical has received accelerated approval from the US Food and Drug Administration (FDA) for its gene therapy, Genglycos (pariglasgene brecaparvovec-opnr), to treat glycogen storage disease type Ia (GSDIa).