Ultragenyx's high burn rate, costly R&D, and recent setbacks increase dilution risk, impacting future profitability. Learn ...

RARE faces regulatory setback as FDA issues a CRL for UX111, delaying approval of the MPS IIIA gene therapy until 2026.

Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a ...

Ultragenyx Pharmaceuticals took another hit when the FDA rejected its gene therapy for patients with a rare, genetic disorder.

Del-zota is designed for patients with mutations amenable to exon 44 skipping, which is just 7% of the overall DMD patient ...

British engineering giant Rolls-Royce announced on Tuesday a $75 million investment to expand its manufacturing operations in ...

Nebius Inc. (NASDAQ: NBIS) soared 17% on Monday after Goldman Sachs initiated coverage with a “Buy” rating and a $68 price target — implying roughly 39% upside from current levels. The investment bank ...

The decision follows the initial approval granted in September 2023, which covered patients aged five years and above.

This report also provides a detailed analysis of the current mucopolysaccharidosis marketed drugs and late-stage pipeline ...