A young Kiwi patient has become the first in the world to be treated with a revolutionary new gene therapy. The University of Auckland neurologist leading the trial – targeting a rare and disabling hereditary disorder with a gene-silencing drug – has likened the young man to “the first person to go to the moon”. It’s one of the latest instances of ground-breaking gene therapies, an approach that treats or prevents diseases by correcting the underlying genetic problem that causes them, being trialed in New Zealand.
Its target is facioscapulohumeral muscular dystrophy (FSHD), which is estimated to affect around one in 8000 people and causes weakness in muscles. The drug ARO-DUX4, made by US therapeutics company Arrowhead, targets muscle cells and silences expression of the DUX4 gene, which causes havoc in the bodies of people with FSHD..
