Fresh MND hope as breakthrough drug approved

THE first new treatment for motor neurone disease in almost three decades has been approved for use in the UK — offering hope to patients.

- By Lucy Johnston SOCIAL AFFAIRS EDITOR

The drug, called Qalsody (tofersen), is the first in Britain to directly target a genetic cause of the disease.

It has been hailed a “genuine breakthrough” but 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.

But experts say the breakthrough demonstrates the potential of a new range of gene-targeted therapies, which are being trialled in other forms of the condition and offer hope to thousands more.

Professor Chris McDermott, a leading consultant neurologist and Sheffield MND Care Centre co-director, said: “This drug is a game changer for many of the patients who have taken it and noticed a steep 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 sufferers.