Ultragenyx sees enough in Angelman
The group buys out its partner Genetx, but inconsistent data spook investors.
Biotech catalysts on the horizon
The second quarter should bring pivotal data for Karuna and key cardiomyopathy results for Alnylam, while Ionis's eplontersen lags in amyloidosis.
Astra gets Wilson disease bonus
ALXN1840 could challenge older copper chelators, but gene therapies are coming.
Sigilon hold hurts ahead of key haemophilia meeting
After a patient develops inhibitors questions emerge about a new approach to treating haemophilia A.