Who is this study for: Patients over 14 with hereditary spastic paraplegia type 5
Enrollment Status: Recruiting
Publish Date: November 23, 2021
Intervention Type: Drug
Study Drug: Evolocumab
Study Phase: Phase 1/Phase 2
Summary: Spastic paraplegia type 5 (SPG5) is a rare subtype of hereditary spastic paraplegia, a highly heterogeneous group of neurodegenerative disorders defined by progressive neurodegeneration of the corticospinal tract motor neurons. SPG5 is caused by recessive mutations in the gene CYP7B1 encoding oxysterol-7a-hydroxylase. This enzyme is involved in the degradation of cholesterol into primary bile acid...