News about IONIS-HTT Rx: a possible future treatment

In December 2017, Huntington’s disease made global headlines for a great reason: scientists had made a breakthrough in the hunt for a treatment. The drug, called Ionis-HTT Rx, is hoped  to lower the amount of huntingtin protein in a person with HD which may slow down, stop or even reverse some of the symptoms of HD.

Ionis-HTT Rx was tested for safety on 40 volunteers over a 27-month period. The trial results were published in a media release on December 11, 2017, with two important announcements:

  1. The drug proved safe for humans, allowing researchers to plan for the next phase of testing: outcomes.
  2. Early indications are that the drug is effective in reducing the amount of huntingtin protein.

What researchers do not yet know is whether or not this reduction through IONIS-HTT Rx treatment will have any effect on people’s symptoms. There is hope that by ‘silencing’ the protein, symptoms may be reduced, stopped or even reversed, but this is not yet known. The next phase of the trial, hopefully starting late in 2018, will investigate this.

Here we are compiling many of the key news stories and articles about the drug IONIS-HTT Rx.

News articles about IONIS-HTT Rx