What is happening inside the body in ALS

In more than 90% of ALS cases, a protein called TDP-43 accumulates abnormally in the neurons in the brain and spinal cord.

However, patients with ALS with SOD1 mutation, the most common familial form of ALS, do not have accumulation of TDP-43, instead have abnormal accumulation of SOD1. These accumulated proteins are the key causes of the disease.


Currently, only two drugs have been approved in the USA and Japan, both of which slow disease progression, but have limited efficacy. Unfortunately, there are no drugs that can fundamentally cure this disease.

Riluzole (Rilutek®)

Edaravone (Radicat®)




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