The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...

This is the first time such results have been achieved in both children and adult patients born with a specific type of ...

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

Simmons, 75, and his wife, Shannon Tweed, purchased an eco-friendly blufftop mansion in September 2024, Realtor.com can ...

The enzyme AMPD1 plays a key role in muscle energy production and normal muscular function. Loss of AMPD1 activity due to ...

This AI system can analyze up to one million DNA letters at once, predicting how tiny changes in noncoding regions trigger ...

A glucose-sensitive nanovaccine delivers CRISPR to tumors, silences immune-suppressing genes, and activates a targeted immune response against cancer.

While humans can regularly replace certain cells, like those in our blood and gut, we cannot naturally regrow most other ...

The KISS rocker picked up the “one-of-a-kind” estate in September. It has 6 bedrooms, 9 baths and sweeping views of the ocean ...

Scientists have gene-hacked mice to produce their own Ozempic-like drugs, paving a path for humans to do so themselves one ...

The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...