Huntington’s Disease: Drug Therapy

Therapeutic target

  • Relief of symptoms

Therapy recommendations

Further notes

  • In a first-in-human clinical trial (Phase I study), an antisense drug showed that it could decrease the disease-causing protein in CSF. The therapy involved intrathecal injections of an oligonucleotide that can bind and destroy the messenger RNA of huntingtingens. The primary endpoint of the study was safety. No serious complications occurred, and none of the patients discontinued treatment prematurely. Meanwhile, a Phase III trial has begun.

Note: Red-hand letter (AkdÄ Drug Safety Mail): contraindications, warnings, and measures to avoid exposure to valproate during pregnancy:

  • In girls and women of childbearing age, valproate should be used only if other treatments are not effective or are not tolerated.
  • Valproate is contraindicated in women of childbearing age unless the pregnancy prevention program is followed.
  • Valproate is contraindicated in epilepsy during pregnancy unless no suitable alternatives are available.
  • Valproate is contraindicated during pregnancy for bipolar disorder and migraine prophylaxis.