The Spastic Paraplegia - Centers of Excellence Research Network (SP-CERN) is a collaborative research consortium dedicated to advancing the understanding, diagnosis, and treatment of hereditary spastic paraplegia (HSP) and primary lateral sclerosis (PLS). Aims of the consortium are to a) perform natural history studies of HSP subtypes, b) discover and validate biomarkers and clinician- and patient-reported outcome measures, c) uncover HSP's molecular pathophysiology and develop rational therapeutic targets, and d) perform sufficiently powered clinical trials. The current pilot study is aimed at enrolling 100 individuals with hereditary spastic paraplegia type 4 (SPG4) or hereditary spastic paraplegia type 5A (SPG5A).
Male or female patients of all ages with a clinical and molecular diagnosis of hereditary spastic paraplegia type 4 (SPG4, SPAST) or hereditary spastic paraplegia type 5A (SPG5A, CYP7B1).
Not having such a diagnosis and/or not being related to such individual.
Boston Children's Hospital
Lead sponsor
Massachusetts General Hospital
Collaborator
Columbia University
Collaborator
University of Miami
Collaborator
University of Michigan
Collaborator
Baylor College of Medicine
Collaborator
University of Texas Southwestern Medical Center
Collaborator
University of Washington
Collaborator
Children's Hospital Medical Center, Cincinnati
Collaborator
Seattle Children's Hospital
Collaborator
University of Iowa
Collaborator