US FDA grants Rare Pediatric Disease Designation to Axovant’s AAV9-based gene therapy, AXO-AAV-GM1 for GM1 gangliosidosis
Axovant Gene Therapies Ltd., a clinical-stage company developing innovative gene therapies for neurological diseases, announced that it has received Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for AXO-AAV-GM1, an...


Recent Comments