Tofersen treatment for rare MND to be subsidized on PBS

A new treatment for a rare form of motor neurone disease (MND) will be available via the Pharmaceutical Benefits Scheme (PBS) starting October 1. The drug, Tofersen, targets patients with a specific SOD1 gene mutation.

In patients with SOD1-related MND, the mutation causes a “toxic gain of function” where the gene attacks and destroys motor neurons in the brain and spine. Professor Dominic Rowe, a clinical neurologist, stated that the therapy works by reducing the amount of toxic protein in motor neurons. This reduction improves the ability of these neurons to survive, which slows the progression of weakness and can extend a patient's lifespan and quality of life.