While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...
Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently getting genes and gene-editing tools where they need to be within cells.
UniQure needs to run another trial to prove its gene therapy "actually helps people with Huntington's disease," said a senior FDA official.
Yentli Soto Albrecht has a rare genetic form of ALS and is working on research at Penn that could one day prove life-saving.
A remarkably small bacterium containing fewer than 500 genes serves as the basis for one of the most detailed digital life reconstructions ever created. Using computer technology, scientists have ...
Pete Docter says Pixar cut the LGBTQ storyline from 'Elio' because 'we're making a movie, not hundreds of millions of dollars of therapy.' ...
We are in the golden age of science and technology, advancements in gene editing and ASO therapies are allowing doctors to deliver targeted, and in some cases one-time, interventions for conditions ...
A gene borrowed from the naked mole rat, an animal famous for extraordinary longevity and disease resistance, helped mice live a little longer and stay healthier, pointing to a surprising biological ...
Market discussion surrounding the company aligns with broader sentiment linked to the nasdaq composite.
Creatures & Corridors is Stranger Things meets Jumanji by way of the Satanic Panic of the 1980s and '90s. Creatures ...
Disease research has always been about understanding tiny pieces of life. Scientists study how cells behave when sickness ...
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