Despite gaps in our understanding of the biology that underlies this neurodegenerative condition, potential treatments are on the horizon.
Nature Outlook |
There is fresh hope for treating Huntington’s disease, an inherited neurodegenerative condition that causes uncontrollable movements, emotional disturbance and the loss of mental abilities. But biological mysteries remain.
This Nature Outlook is editorially independent. It is produced with third party financial support. About this content.
Even as new treatments loom, researchers are still trying to understand what goes wrong inside the cells of people with the genetic disorder.
With a family history of the condition, Mark Newnham makes a tough decision to find out what his future could hold.
Antisense oligonucleotides are providing researchers and patients with fresh hope of targeting the condition’s genetic cause.
Gene editing offers the prospect of curing the inherited neurodegenerative condition in a single dose.
Roche in partnership with Ionis Pharmaceuticals is committed to supporting the needs of the community living with Huntington’s disease (HD) and developing treatments for those affected by this devastating disease. We invited journalist and HD patient advocate Charles Sabine to give his perspective at an event organized at Roche in recognition of World Rare Disease Day 2018.
Although it usually affects people in middle age, the inherited neurodegenerative condition can also develop in children and teenagers.
Determining suitable endpoints for clinical trials will help researchers to develop better treatments for the progressive neurological condition.
Although potential treatments are now entering the pipeline, the molecular cause and progression of Huntington’s disease continue to elude researchers.