The Expanding Landscape of Therapeutic Targets in ALS Clinical Trials

Nicholas Maragakis, director of the Johns Hopkins ALS Center for Cell Therapy and Regeneration Research, covers some of the major, clinically relevant pathways of ALS and clinical trials that target those pathways with innovative research design. #ALSTherapy #JohnsHopkins www.hopkinsmedicine.org/neuro...
Chapters:
7:43 Gene therapy targets
15:16 Nucleocytoplasmic transport
17:37 Neuroinflammatory pathways
19:58 Immune-mediated ALS therapies
24:17 Highlight of platform trial design
28:55 Cellular stress
33:09 Muscle targets
35:18 Stem cell therapies
37:44 ALS clinical trials unit
39:30 Questions