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 ...

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

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

Nanoscope Therapeutics submitted the first modules of a biologics license application for MCO-010, an investigational gene ...

While the complete response letter derails a near-term clearance, one analyst described the rejection as more of “a speed ...

A rare metabolic condition affecting children may have to wait longer for a potential one-time treatment as Ultragenyx ...

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

Most genes are ancient and shared across species. But a small subset of genes are relative newcomers, spontaneously emerging ...

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 ...

A fire on July 13 destroyed Betty Gene’s restaurant, the Community Market, and nearby offices in Madison, but no injuries ...

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