A groundbreaking new treatment for motor neurone disease (MND) has been approved for use in the UK, offering fresh hope after decades of despair.
But campaigners warn that patients may still have to wait for access on the NHS.
The drug, called QALSODY (tofersen), is the first in Britain to directly target a genetic cause of the disease.
It is being hailed as a “genuine breakthrough” - the first new treatment for MND in almost 30 years.
The new drug will only help those with a rare inherited type of MND caused by a fault in the SOD1 gene - thought to affect fewer than 60 people in the UK.
However experts say the breakthrough demonstrates the potential of a new era of gene-targeted therapies, which are currently being trialled in other forms of MND offering hope to thousands of patients diagnosed each year.
Professor Chris McDermott, a leading consultant neurologist and Sheffield MND Care Centre Co-Director said: “This drug is a gamechanger for many of the patients who have taken it who have noticed a step change in the way their disease progresses with some also noticing improvement in their function. Some of these patients would not be alive now were it not for this drug.”
He added: “This technology is a major milestone for all MND sufferers and is now being used in trials to target other key genes and proteins for other forms of MND.
