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Oral drugs, the new frontier in fighting the cause of Huntington's disease

PTC Therapeutics recently published an article in the prestigious journal Nature Communications describing the functioning of a series of small molecules, that can be taken orally, and are able to distribute themselves in the brain and also in the rest of the human body.


Reducing levels of huntingtin, the protein that, when mutated, causes Huntington's, was the first strategy designed to directly target the cause of the disease.

However, the current approaches  have some limitations, as the molecules under study:

  • Are unable to cross the blood-brain barrier, and therefore require invasive administration procedures, such as a lumbar puncture or a surgery;
  • Are not distributed in the whole brain, but only in same areas;
  • Do not reduce huntingtin in peripheral tissues.

The molecules studied by PTC, on the opposite, seem to have all the features missing so far, because they:

  • Reduce huntingtin in the brain AND in the body
  • Can be taken orally
  • Are selective for huntingtin
  • Their effects are reversible 

The very interesting study by PTC Therapeutics was recently published on Nature Communication and it is very well described on the HDBuzz website.