Ultragenyx's Strong Pipeline And Valuation Concerns: A Balanced Perspective
RAREUltragenyx Pharmaceutical(RARE) Seeking Alpha·2024-08-22 22:22

Andrzej Rostek Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) develops innovative therapies for rare and ultra-rare genetic diseases. Its commercial portfolio includes Crysvita, Mepsevii, Dojolvi, and Evkeeza, which address unmet medical needs in conditions like X-linked hypophosphatemia [XLH], Mucopolysaccharidosis VII [MPS VII], long-chain fatty acid oxidation disorders [LC-FAOD], and homozygous familial hypercholesterolemia [HoFH]. Additionally, RARE's pipeline targets severe diseases such as osteogenesis ...