Ultragenyx Pharmaceutical Inc.
NEWS
It was yet another busy week for clinical trial news, with a range of studies for COVID-19, various cancers, fungal infections and other indications.
Ultragenyx and Mereo celebrated their first patient dosed in a Phase II/III study investigating the use of setrusumab as a treatment for osteogenesis imperfecta (OI).
When Sekar Kathiresan’s brother unexpectedly died from a heart attack, he changed the course of his life to found a company with a novel medicine that may prevent them.
Several CEOs have stood out from their peers throughout the past year, winning recognition from the Healthcare Technology Report. BioSpace looks at five of these leaders.
The Glycogen Storage Disease Program at Connecticut Children’s and UConn Health reported success in a Phase I/II trial of a gene therapy for GSD-Ia.
GeneTx Biotherapeutics and Ultragenyx Pharmaceutical’s jointly prepared treatment for Angelman syndrome have been provided a green single by DSMB recently.
Collaboration is key to faster breakthroughs. The NIH, FDA, and 15 private organizations have announced they are joining forces for the sake of the 30 million Americans suffering from a rare disease.
It was yet another busy week for clinical trial news. Here’s a look.
Redosing is a large and growing concern for gene therapy developers and regulators.
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