Hereditary Spastic Paraplegia Type 47
📧 Sign up to get email alerts when something new happens for Hereditary Spastic Paraplegia Type 47.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS research · 2026-03-18Clinical Trial Update: Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia
A registry and natural history study for early onset hereditary spastic paraplegia is currently recruiting.
-
CLINICAL_TRIALS trial_result · 2025-04-28Clinical Trial Update: Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47) [Phase 1, Phase 2]
Phase 1/2 clinical trial assessing safety and efficacy of BFB-101, an AAV9-delivered AP4B1 gene therapy, for AP4B1-related Hereditary Spasti…