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Parallel Session 3411

The New Era of Neurometabolic Movement Disorders

Tuesday, October 7, 2025
16:30 - 18:00 | Room TBD
Translational

This session is focused on advances in mechanistically-driven targeted precision therapies, that are either in preclinical development, clinical trial or established therapies for children and adults with complex neurometabolic diseases.


Chairs:
Manju Kurian, United Kingdom
Niccolo Mencacci, USA

Presenters:

Precision therapies for NBIAs
Susanne Schneider, Germany

Small Molecule Therapies
TBD

Genetic Therapies
Toni Pearson, USA

CSPC Liaisons:
Manju Kurian, United Kingdom

Learning Objectives
At the conclusion of this session, participants should be better able to:

  1. Describe the different precision medicine strategies for treating Neurodegeneration with Brain Iron Accumulation disorders
  2. Describe the various precision approaches including small molecule therapies for neurometabolic disorders
  3. Discuss the role of genetic therapy in inherited monogenic neurometabolic disorders, including DNA and RNA therapies

Recommended Audience
Clinician / General Neurology
Fellow / Resident / Student
Health Professional (non-physician)
Industry
Researcher / Basic Science

Education Level
Advanced / Expert
Beginner / Foundational
Intermediate / Experienced

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