Breakthrough in fight against Huntington's disease
Results from a ground-breaking drug trial have offered new hope to people with Huntington's disease.
The experimental drug, injected into spinal fluid, was shown to lower levels of the harmful protein responsible for the devastating inherited brain disorder.
IONIS-HTTRx is the first pharmaceutical agent designed to target the root cause.
A total of 46 patients with early-stage Huntington’s disease took part in the trial conducted at nine centres in the UK, Germany and Canada.
The study confirmed the treatment worked as hoped and was well tolerated by the participants.
Huntington’s disease is a progressive neurological disorder that normally appears in adulthood and causes involuntary movements and dementia.