Del-zota is designed for patients with mutations amenable to exon 44 skipping, which is just 7% of the overall DMD patient ...

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

Panelists discuss how payers seek good return on investment when evaluating expensive gene therapies, creating potential friction when innovative treatments come at significant costs, requiring ...