Roundtable Discussion: Advancing Combination Therapies in ALS: Integrating Small Molecules & Genetic Medicines for Greater Therapeutic Impact
- Scientific rationale for combining small-molecule neuroprotective agents with genetic medicines to target multiple ALS disease pathways, including RNA dysregulation and motor neuron degeneration
- Lessons from preclinical and early clinical PrimeC data showing enhanced efficacy and biomarker modulation with multi-component combination strategies versus monotherapies
- Regulatory considerations for combination therapies, including trial design, biomarker endpoints, and dual-mechanism product development
- Patient and advocacy perspectives on the benefit–risk profile of oral multi-target combinations, and opportunities for industry collaboration to accelerate adoption