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 ...

Nearly 11 years after cancer treatments, an Alexandria resident continues to raise funds for cancer research amid another ailment.

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 ...

complicated hereditary spastic paraplegia, and select hereditary metabolic neuropathies. Conclusions Diverse inherited neuropathy patients without genetic discovery by candidate gene testing have a ...