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ALS research is entering a pivotal new era, driven by advances in innovative research, clinical trial models and biomarkers that are accelerating the development of therapies that aim to slow progression and improve quality of life. Among these, Mass General Brigham’s HEALEY ALS Platform Trial stands out as a transformative approach, enabling multiple investigational therapies to be evaluated efficiently within a single, coordinated framework.
In this webinar, experts from the Healey & AMG Center and Neurizon will discuss the design and impact of platform trial models, and the introduction of Neurizon’s investigational therapy NUZ-001 under regimen “I”. NUZ-001 targets TDP-43 aggregation and has demonstrated long-term safety with encouraging clinical and biomarker signals in Phase 1 and OLE studies.
Join us as we explore:
- Structure and scientific rationale of HEALEY ALS Platform Trial
- How adaptive trial models accelerate patient access and robust data generation
- Scientific rationale for NUZ-001, including its TDP-43-targeted mechanism
- Key insights from Phase 1/OLE studies: safety, functional outcomes, respiration, biomarkers
- Opportunities for collaboration across academia, industry, and patient communities
Offered Free by: Neurizon
See All Resources from: Neurizon
