UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms
While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...
UniQure needs to run another trial to prove its gene therapy "actually helps people with Huntington's disease," said a senior FDA official.
A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...
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 ...
BioAnalysis launches BASIS 2.0, its next-gen GMP-compliant AUC software for gene therapy. Higher throughput, one-click ...
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 ...
Eli Lilly advances CRISPR collaboration with Scribe Therapeutics for neurological and neuromuscular disorders; European ...
Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, hemophilia and, recently, Huntington's disease, a fatal neurological ...
Katie Jackson desperately wants a new treatment for Huntington’s disease. Her husband died from the devastating brain disorder. And because the disease runs in families, her three children have a 50% ...
The DNA foundation model Evo 2 has been published in the journal Nature. Trained on the DNA of over 100,000 species across ...
Flight attendant Kurt Hanson shares three decades of high altitude glamour, hidden secrets, personal tragedy, and an ...
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