While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...

For the first time, scientists have successfully treated someone suffering from Huntington's disease, a fatal genetic illness ...

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 ...

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 ...

By Michael Erman and Puyaan Singh March 5 (Reuters) - A senior FDA official called UniQure's experimental treatment for ...

In a groundbreaking first, a gene therapy in clinical trials has slowed the progression of Huntington's disease, a rare genetic disorder in which toxic bits of protein cause brain cells to malfunction ...

Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...

UniQure’s AMT-130 is a gene therapy to reduce levels of the Huntingtin protein in the brain. ・The FDA recently said that data ...

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 ...

Subtle changes in the brain, detectable through advanced imaging, blood and spinal fluid analysis, happen approximately twenty years before a clinical motor diagnosis in people with Huntington's ...

UniQure needs to run another trial to prove its gene therapy "actually helps people with Huntington's disease," said a senior FDA official.