41 recruiting studies matching your filters
The goal of this clinical trial is to help understand spinal cord excitability in children with cerebral palsy compared to neurologically typical children. The…
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to s…
The goal of this clinical trial is to learn if targeted medical therapy will improve symptoms and quality of life in patients with angina and non-obstructive c…
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…
This study uses medical records that allow retrospective data extraction of clinical manifestation to assess the natural history of HPDL mutations