While developing a drug called branaplam for patients with SMA, the pharmaceutical company Novartis discovered that it could hold promise for people with HD. The FDA has granted a special status called Orphan Drug Designation.
The SIGNAL study did not meet its key clinical goals for #HuntingtonsDisease to slow or improve HD symptoms, but the results are still informative for the HD community and other fields.
CAG repeats expand in some parts of the body and brain as people with HD get older, a phenomenon known as somatic instability. Learn more about how researchers are exploring somatic instability and DNA repair to design therapies for HD.
Scientists use human fetal tissue to look at HD brain development. But what do developmental changes mean when symptoms don’t occur until decades after birth?
A recent series of studies on the gene-editing method CRISPR have raised concerns about the suitability of this technology for the treatment of genetic illnesses such as Huntington's disease
Scientists recently used an antihistamine to quiet dopamine messages in the brain and treat HD-like symptoms in mice. But beware the hype suggesting that allergy medicines could be used to slow down HD.
Because HD causes a loss of neurons in the brain, some researchers are exploring ways to replace them. Working with HD mice, scientists recently showed that supportive brain cells called glia can be coaxed into becoming new neurons.
We know that HD-related changes can occur many years before symptom onset, but how early do those changes begin? A team of researchers set out to determine that with a new comprehensive study in pre-manifest HD young adults.
Another clue about the normal function of the huntingtin protein; a team of scientists has recently found that huntingtin seems to play an important role in repairing damaged nerve cells
COVID-19 update: what does it mean for HD families, how does it impact HD research, and how has it changed the way science works?
A collaborative team of scientists from Canada and Japan have identified a small molecule which can change the CAG-repeat length in different lab models of Huntington's disease. #HuntingtonsDisease #DrugDiscovery