Organizer
CNS Research Committee
Daniel Calame, MD, PhD
Baylor College of Medicine
Description
The hereditary spastic paraplegias (HSP) are a group of over 80 different monogenic diseases that present with progressive spasticity and weakness. This webinar will introduce childhood-onset HSP on clinical and molecular levels. We will focus on four key areas: 1) A framework to the challenges and opportunities of studying rare diseases; 2) Childhood-onset HSP in the differential diagnosis of cerebral palsy; 3) Establishing clinical trial readiness for HSP, and 4) the development of molecular mechanism-based treatments including molecule drugs and AAV-based gene replacement.
