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 ...

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

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 ...