Sigma-1 Receptor Agonism in ALS: Why, How & When?
- Explaining why sigma-1 receptor agonism is an important target for ALS, including human genetic data
- Providing in vitro, in vivo and human data to support this
- How this data and prior Phase 2 data encouraged design of Phase 3 pivotal trial, including novel endpoints such as speech
- Timelines, milestones and potential outcomes, and providing insights from ongoing NIH-funded expanded access program (EAP)
- Similar mechanisms may highlight sigma-1 agonism for other neurodegenerative disorders, such as FTD