Tofersen, has been designed to work by stopping the production of abnormal SOD1 protein that is toxic to motor neurons. A previous clinical trial called VALOR showed some hints that Tofersen was having some effect in reducing the amount of SOD1 protein in people with MND however, no significant difference between those taking the medicine and those taking the placebo was shown on the primary endpoint (ALSFRS-R). Following this, an open label extension phase commenced to see if taking the treatment for a longer time led to more of an effect. This new data was presented at ENCALS and showed results leading to cautious optimism that the treatment may be effective if given for a longer time. Click here to read more.
