Ultragenyx

News

Ultragenyx gets FDA nod for first Sanfilippo A gene therapy

Patients in the US with Sanfilippo syndrome type A have their first FDA-approved therapy, Ultragenyx Pharma's $3.9m one-shot gene therapy Fayuvi.

News

Ultragenyx to slash costs after Angelman trial failure

Ultragenyx's therapy for the rare genetic disorder Angelman syndrome, apazunersen, has flunked a late-stage trial, crashing its share price.

News

Ultragenyx wins FDA approval for first treatment targeting r...

Ultragenyx secured its first gene therapy approval, the FDA clearing GENGLYCOS as the first treatment designed to address underlying cause of GSDla.

News

Ultragenyx, Mereo's brittle bone drug flames out in trials

Two phase 3 trials of Ultragenyx and Mereo's sclerostin inhibitor setrusumab in osteogenesis imperfecta missed their primary efficacy objectives.

R&D

This week in clinical trials – 27th to 31st May

In this week’s clinical trials round-ups, we look at life sciences research and development announced during the week of 27th to 31st May, from early-stage onwards.

News

Judge says Ultragenyx must face Henrietta Lacks estate suit

An attempt by Ultragenyx to have a lawsuit filed by the family of Henrietta Lacks dismissed before it could be heard has failed.

Ultragenyx gets FDA nod for first Sanfilippo A gene therapy

Ultragenyx to slash costs after Angelman trial failure

Ultragenyx wins FDA approval for first treatment targeting r...

Ultragenyx, Mereo's brittle bone drug flames out in trials

This week in clinical trials – 27th to 31st May

Judge says Ultragenyx must face Henrietta Lacks estate suit

Lily gets FDA nod for SERD-CDK 4/6 combo in breast cancer

Discover a new era of healthcare panel engagement: Grounded ...

Ultragenyx

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