New Therapeutic Target Identified for ALS

Scientists have identified a novel therapeutic approach for the most frequent genetic cause of Amyotrophic lateral sclerosis (ALS).

The ALS Association and the Packard Center Partner to Develop Animal Model Systems for Most Common Cause of Familial ALS

“Following the discovery of the most abundant genetic cause of ALS, we have initiated a drug development approach to selectively destroy the ALS-causing product of the mutated gene…”