The agency's decision is based on results from a clinical trial in which the treatment improved hearing in 80 percent of the ...
Astellas Pharma has done some spring cleaning of its pipeline, dropping two phase 1 candidates and slamming the brakes on a ...
Intellia Therapeutics, a leading biotechnology company, announced that its CRISPR-based treatment for a rare swelling ...
The treatment, developed by Regeneron Pharmaceuticals, is for a very rare form of deafness. But it represents a medical ...
Specialty drugs are an increasing worry for health plans and employers as new and innovative therapies enter the U.S. market ...
In vitro data demonstratedself-complementary AAV9 (scAAV9) enabled ~30-fold higher MeCP2 protein expression compared to single-stranded AAV9 ...
Topline results from the pivotal HAELO trial demonstrate that a single infusion of lonvo-z significantly reduces HAE attacks and the need for chronic prophylactic therapy.
Learn about the groundbreaking gene therapy for sickle cell disease at Manning Family Children's Hospital in Louisiana, ...
Researchers say a gene therapy allowed deaf children and adults as old as 32 to hear for the first time. The benefits have ...
A collaboration of scientists at the University of Manchester and the University of Birmingham has explored a more effective ...
The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time ...
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