Identifying novel therapeutic targets related to TDP-43 loss
Despite the diversity of ALS, almost all patients have one thing in common: the protein TDP-43 stops performing its normal functions in the nucleus, leading to disrupted splicing and decreased expression of many genes.
The Impact of Gene Fusions on ALS Disease Progression
We predict that ALS-specific gene fusions may improve many aspects of clinical care as already
demonstrated in the field of oncology and are now working to understand how the fusions effect ALSdisease progression.
Investigating Therapeutic Options to Increase STMN2 Levels
Researchers at the Sean M. Healey & AMG Center for ALS, along with their colleagues, demonstrated that pharmacological compounds and gene therapy that increase the levels of STMN2 can restore the ability of neurons to grow axons after injury.
Discovering subtypes of ALS
When the root cause of ALS is known, there are now paths to therapy, as has been shown by the recent development of tofersen for people whose disease results from the SOD1 gene.
