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Goals, Mission & Vision

Goals

The goal of the SP-CERN is to create clinical trial readiness, support therapy development, and increase access to high-quality healthcare for individuals with hereditary spastic paraplegia (HSP). Physician-scientists and their multidisciplinary teams from different research centers across the United States will collaborate to develop a framework for translational research for HSP that will ultimately improve the lives of children and adults affected by HSP.

  • Perform natural history studies of HSP subtypes
  • Discover and validate biomarkers and clinician- and patient-reported outcome measures
  • Uncover HSP’s molecular pathophysiology and develop rational therapeutic targets
  • Perform sufficiently powered clinical trials
  • Bring together a community of individuals affected by HSP
  • Establish a shared clinical database, biobank of biospecimen samples, and a central repository of genomic data
  • Train the next generation of clinicians and scientists
  • Facilitate the clinical testing of novel therapies Increase access to high-quality healthcare for HSP
  • Foster international collaboration
  • Synchronize and harmonize research worldwide

Create the central infrastructure of SP-CERN by developing three central core units:

  • Establish a Central Administrative Core
  • Establish a Career Enhancement Core
  • Establish a Pilot/Feasibility Project Core

Conduct three Clinical Research Projects to meet critical unmet needs in HSP research:

  • Establish a clinical trial readiness platform for HSP
  • Develop genomic resources and studies for SP-CERN
  • Investigate digital surrogates for disease progression in HSP

Contribute to the RDCRN and Rare Disease Community:

  • Build a collaborative network with patient advocacy groups
  • Harmonize the SP-CERN study protocol internationally
  • Contribute to the RDCRN’s broader mission

SP-CERN Mission Statement

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).