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Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP)
The Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP) is focused on gathering longitudinal clinical data as well as biolog…
Hereditary Spastic ParaplegiaSPG47SPG50+8 more
Boston Children's HospitalNCT04712812
Phase 1
Safety and Efficacy of AAV9/AP4B1 For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of…
HSPHereditary Spastic ParaplegiaHereditary Spastic Paraparesis+10 more
BlackfinBio LtdNCT06948019