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

Animal statues can be seen across downtown Pella, bringing a children’s story to life while also honoring special community ...

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

Acumen and JCR aim to develop a product leveraging Acumen's amyloid beta oligomer-selective antibody expertise and JCR's transferrin-receptor-targeting blood-brain barrier-penetrating technologyPrecli ...

Preclinical candidate data expected in early 2026, with exclusive option for Acumen to develop up to two development candidates for the treatment of Alzheimer’s disease Agreement follows more than one ...