New treatment offers hope for sufferers
A new treatment called Tofersen has been approved for subsidy in Australia to treat a rare form of motor neurone disease caused by a specific SOD1 gene mutation. The therapy works by reducing toxic proteins that destroy motor neurons, offering hope for slowing disease progression.
Why it matters
This represents a significant medical breakthrough for patients with a rare, fatal genetic condition, providing access to life-extending therapy.
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Share A A A Over the past four decades, Nicole Lynch has watched motor neurone disease strip her family members of their agency and rob years from their lives.
In 1995, her cousin was diagnosed with MND. Not long after, so was another cousin. Her mother died from MND at the age of 51.
Lynch will be one of the beneficiaries of the PBS listing of a new treatment for a rare form of ALS. James Brickwood “It’s awful … The worst thing about it is knowing what’s going to happen,” the 54-year-old said.
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