In particular we are investigating the involvement of axonal transport in diseases including motor neurone disease (MND) and hereditary spastic paraplegia (HSP). In addition we are developing and ...

Hereditary spastic paraplegia, or HSP ... animal models is an indispensable part of developing and testing any new treatments before they're offered to humans. But scientists had historically ...

The pipeline comprises BFB-101, an IND stage AP4B1 replacement AAV gene therapy for Hereditary Spastic Paraplegia, sub-type 47 - an ultra-rare genetic neurological disease for which no treatment ...