The Glycogen Storage Disease Program at Connecticut Children’s and UConn Health reported success in a Phase I/II trial of a gene therapy for GSD-Ia. The gene therapy is developed with Ultragenyx and is dubbed DTX401.
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Led by new CEO Dr. Yvonne Greenstreet, Alnylam Pharmaceuticals has a stated ambition to transition the RNA interference therapeutics trailblazer to a top 5 biotech – measured by market capitalization – within the next five years.
Visionary tech magnate Elon Musk is taking his neurotech company to the next step toward in-human trials of his implantable brain-machine interface.
New coronavirus cases are falling in parts of the United States hit hardest by the fast-spreading Omicron variant, according to a Reuters analysis of public health data, offering an early indication the virus might once again be in retreat.
Alzheimer’s patient groups, disappointed by Medicare’s plan to sharply limit coverage of new drugs for the brain-wasting disease, are planning publicity and lobbying campaigns to protest a proposal they say could delay their use for 10 years.
Researchers have been working to develop approaches to curing Type 1 diabetes by transplanting healthy islet cells into the pancreas. Investigators at Northwestern University have developed a technique to make the immunomodulation effect of immunosuppressive drugs more effective.
The U.S. Department of Homeland Security (DHS) announced it is requiring that non-U.S. essential workers such as truck drivers and nurses who are crossing land borders be fully vaccinated against COVID-19, effective January 22.
The Omicron variant, which is spreading far faster than previous versions of the coronavirus, is not likely to help countries achieve so-called herd immunity against COVID-19 – in which enough people become immune to the virus that it can no longer spread – leading disease experts say.
Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases and White House chief medical advisor, said they expect the U.S. Food and Drug Administration will authorize the Pfizer-BioNTech vaccine for children under the age of 5 years in the next month.
A small preliminary laboratory study has shown that levels of Omicron-neutralizing antibodies of people vaccinated with Russia’s Sputnik V vaccine did not decline as much as those who had Pfizer shots.