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Antisense therapy promises to slow Huntington鈥檚 disease

Drug injections into the spine sabotaged production of mutant protein that destroys brain cells

AN INCURABLE inherited illness that kills many of those affected by their 40s may be slowed by a novel drug injected into fluid surrounding the spine and brain.

Although the in 46 people with Huntington鈥檚 disease in the UK, Germany and Canada were only preliminary, independent neurologists were still cautiously optimistic.

The drug is designed to stop production of the huntingtin protein, which is mutated in those with the condition and kills brain cells, causing gradual loss of mobility and brain activity.

Before it can be produced, the treatment intercepts and destroys RNA that carries the instructions to make the protein. It is the first time a drug has cut levels of the toxic version of huntingtin in the nervous system of people. A larger trial will be needed to see if this slows disease progression.

This article appeared in print under the headline 鈥淗untington鈥檚 hope鈥

Topics: Diseases / Drugs