Ultragenyx Pharmaceutical Inc.
NEWS
BioSpace spoke with several leaders to gather their thoughts and predictions for the new year. Here is the first installment of this two-part series.
The buyout comes on the heels of promising Phase I/II results from GTX-102, an antisense oligonucleotide candidate being developed to treat Angelman syndrome.
ION582 is uniquely formulated using RNA technology to provide relief to patients with Angelman syndrome, a rare disorder for which limited treatment options are available.
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.
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