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.

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

Carlsmed Inc. launched its IPO at a price of $14 to $16 per share for 6.7 million shares, which would raise just over $100.5 ...

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 ...