Two new approaches to silence the SOD1 gene that causes ALS were announced today, signaling the beginning of personalized medicine in ALS. Healey Center investigators were instrumental in both studies. 

Phase 1/2 Study of Tofersen for SOD1 Amyotrophic Lateral Sclerosis

Full press release from Washington University School of Medicine

Full press release from Biogen

Article in the New England Journal of Medicine

SOD1 Gene Expression with Adeno-associated Virus and MicroRNA in Familial ALS

Full press release from UMass Medical School

Article in the New England Journal of Medicine