Adam Feuerstein ✡️(@adamfeuerstein):I spoke with Lauren Holder, a Huntington's patient and advocate, about this week's $QURE AMT-130 gene therapy clinical update. “I’m not surprised by the four-year results because this isn’t a cure,” Holder told me. “This is a slowing-of-disease-progression treatment, and so I would expect there’s still decline going on, and we’re going to see that on testing, and that doesn’t mean it’s not working.” Holder is a lot less pleased with the proposed design of UniQure's confirmatory study, which will require some patients to be randomized to a standard treatment arm. "It’s not ethical,” she said. “You’re requiring patients to not receive any treatments for three years or longer, and to not be able to participate in other research. To me, it’s not ethical. To the community, it’s not ethical, and I really feel we need to have a better option.” Read more:

2026.10.01 13:19

I spoke with Lauren Holder, a Huntington's patient and advocate, about this week's $QURE AMT-130 gene therapy clinical update. “I’m not surprised by the four-year results because this isn’t a cure,” Holder told me. “This is a slowing-of-disease-progression treatment, and so I would expect there’s still decline going on, and we’re going to see that on testing, and that doesn’t mean it’s not working.” Holder is a lot less pleased with the proposed design of UniQure's confirmatory study, which will require some patients to be randomized to a standard treatment arm. "It’s not ethical,” she said. “You’re requiring patients to not receive any treatments for three years or longer, and to not be able to participate in other research. To me, it’s not ethical. To the community, it’s not ethical, and I really feel we need to have a better option.” Read more: