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Neurogenetics Program Patient Registry: Clinical and Genetic Diagnosis, Natural History Study, Translational Research and Biorepository
The objective of this project is to develop a Neurogenetics patient database and bio repository - which includes clinical information regarding history, physic…
Neurogenetic Disorders
University of PittsburghNCT02995538
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