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 ...

In a small trial, a gene therapy injected into the brain slowed the disease by 75 percent over three years. Huntington’s disease is extremely cruel. Symptoms start with random, uncontrollable twitches ...

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 ...

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 ...

Huntington’s disease occurs because of a defective gene. It’s a condition that gets worse over time, and there is currently no cure. Thus, one area of research involves seeking out treatments for the ...

Accordingly, numerous advocacy groups gather in DC to lobby lawmakers about rare disease research, treatments and potential cures. Such rare diseases that generate advocacy interest include Lou Gehrig ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...