Researchers have gained impportant new insights into Huntington's disease-a rare genetic, neurodegenerative disorder... | ...

Treatments that target a fragment of the mutant protein that causes Huntington's disease might be more effective than ...

An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and ...

Huntington's disease is a devastating brain disorder that slowly robs people of movement, memory, and personality. It is ...

While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be considered for approval, experts believe there is an alternate path forward for ...

For decades, so many families have watched loved ones suffer from Huntington's disease, a neurodegenerative condition that progressively worsens over time. But now, there is finally a light at the end ...

For the first time, scientists have successfully treated someone suffering from Huntington's disease, a fatal genetic illness with no cure.

Findings from preclinical studies could point to therapeutic strategies for Huntington’s disease, as well as other neurodegenerative disorders and potentially cancer.

UCL and uniQure report the first treatment to slow Huntington’s disease with gene therapy AMT-130. High-dose patients had 75% less progression over three years compared with standard care. uniQure ...

ST. LOUIS — Scientists may have reached a breakthrough in the fight against Huntington’s disease, a rare and fatal inherited brain disorder with no cure. For the first time, a gene therapy has been ...

PTC518 was granted Fast Track designation based off strong 12-month interim data on safety and efficacy. The Food and Drug Administration (FDA) has granted Fast Track designation to PTC518 for the ...

The first signs of Huntington’s disease are often subtle changes in coordination, such as clumsiness or trouble with balance. You may also begin to notice changes in thinking, such as difficulty ...