Plenary Session 4205

MDS Keynote Lecture and Clinical Breakthroughs

Wednesday, October 8, 2025
8:00 - 09:30 | Ballroom

Chairs:

Ron Postuma, Canada
Michael Okun, USA

Presenters:

Constructing and deconstructing the human nervous system to study development and disease
Sergiu Pasca, USA

Risvodetinib treatment drives clearance of alpha-synuclein pathology in untreated Parkinson’s disease: a randomized Phase 2 Trial
Milton Werner, USA

Safety, tolerability, pharmacokinetics and pharmacodynamics of VTX3232, a CNS-penetrant NRLP3 inhibitor, in participants with early-stage Parkinson’s disease
Mark Forman, USA

Phase 1/2a clinical trial of hESC-derived dopaminergic progenitors in Parkinson’s disease
Han Kyu Na, South Korea

Phase I/II Gene Therapy Study of AMT-130 Slows Huntington’s Disease Progression at 36 Months
Deborah Hall, USA

Expert Panelists
Lorraine Kalia and Susan Fox, Canada

CSPC Liaisons:

Maria Stamelou, Greece

Learning Objectives

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

1. Understand the generation and applications of human stem cells models to study neurological and psychiatric disease
2. Understand the creation of regionalized neural organoids and circuit formation in assembloids
3. Provide examples of disease models and therapeutic development for specific genetic disorders

Recommended Audience

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

Education Level

Advanced / Expert
Beginner / Foundational
Intermediate / Experienced

Focus

Undefined