Ultragenyx

Ultragenyx-Mereo's brittle bone drug sustains fracture rate cut in midphase follow-up
Fierce Biotech
Wed, 06/12/24 - 11:49 am
Ultragenyx
Mereo BioPharma
brittle bone disease
clincial trials
setrusumab
Ultragenyx Pharmaceutical Says Glycogen Storage Disease Treatment Met Endpoints
Marketwatch
Fri, 05/31/24 - 10:46 am
Ultragenyx
clinical trials
DTX401
glycogen storage disorder
Ultragenyx says Angelman therapy is working, but safety questions remain
BioPharma Dive
Tue, 04/16/24 - 07:49 am
Ultragenyx
Angelman Syndrome
GTX-102
Family of Henrietta Lacks sues Ultragenyx over use of HeLa cell line
BioPharma Dive
Sun, 08/13/23 - 02:25 pm
Henrietta Lacks
Ultragenyx
legal
Roche drops antisense drug after seeing efficacy data, leaving Biogen, Ionis and Ultragenyx to duke it out
Fierce Biotech
Fri, 06/23/23 - 09:46 am
Roche
Angelman Syndrome
antisense
Biogen
Ionis Pharmaceuticals
Ultragenyx
rugonersen
mRNA non-vaccine therapeutics field set to reach $2 billion by 2028
Clinical Trials Arena
Tue, 11/8/22 - 10:54 am
MRNA
drug development
Moderna Therapeutics
Ultragenyx
Omega Therapeutics
BioNTech
cancer
OTX-2002
Unpacking Royalty Pharma
EP Vantage
Mon, 07/18/22 - 10:24 am
Royalty Pharma
Ultragenyx
Ultragenyx strikes deal with struggling Abeona for rare disease gene therapy
BioPharma Dive
Tue, 05/17/22 - 10:30 pm
Ultragenyx
Sanfillipo Syndrome
gene therapy
Abeona Therapeutics
Regeneron hands off ex-US rights for cholesterol disorder drug to rare disease specialist Ultragenyx
Endpoints
Fri, 01/7/22 - 10:36 am
Regeneron
evinacumab
Ultragenyx
Ultragenyx granted go-ahead to up doses for Angelman antisense therapy that stalled after safety issues
Fierce Biotech
Wed, 01/5/22 - 10:32 pm
Ultragenyx
GeneTx Biotherapeutics
Angelman Syndrome
clinical trials
antisense
GTX-102
The "Rare Disease Company Coalition" Launches to Share the Unique Challenges and Promise of Rare Disease Therapy Development to Support Continued Progress and Patient Access
Yahoo/Businesswire
Thu, 05/13/21 - 12:04 pm
Rare Diseases
Rare Disease Company Coalition
drug development
drug discovery
Acceleron
Aeglea BioTherapeutics
Agios Pharmaceuticals
Alnylam
Harmony Biosciences
Orchard Therapeutics
Orphazyme
Sarepta Therapeutics
Taysha Gene Therapies
Ultragenyx
Ultragenyx earns 2 rare disease drug approvals—and a validated commercial strategy
Fierce Pharma
Fri, 01/29/21 - 10:00 am
Ultragenyx
FDA
Crysvita
tumor-induced osteomalacia
Kyowa Kirin
Dojolvi
Biotech Mereo BioPharma pens $50M Ultragenyx deal for rare bone disease drug
Fierce Biotech
Fri, 12/18/20 - 11:41 am
Mereo BioPharma
Ultragenyx
setrusumab
osteogenesis imperfecta
Ultragenyx pauses antisense trial after patients lose ability to walk
Fierce Biotech
Tue, 10/27/20 - 10:40 am
Ultragenyx
clinical trials
GTX-102
antisense
Ultragenyx Pharma Wins FDA’s First Drug Nod for Rare Metabolic Disorder
Xconomy
Tue, 06/30/20 - 11:35 pm
Ultragenyx
FDA
long-chain fatty acid oxidation disorders
Dojolvi
Ultragenyx's Crysvita scores FDA nod in 2nd ultra-rare bone disorder
Fierce Pharma
Thu, 06/18/20 - 08:31 pm
Ultragenyx
Kyowa Kirin
Crysvita
FDA
tumor-induced osteomalacia
What Sanofi Might Buy With Its Regeneron Windfall
Barron's
Wed, 05/27/20 - 11:01 am
Sanofi
Regeneron
M&A
gene therapy
Ultragenyx
Bluebird Bio
Ultragenyx shares jump on 'better than expected' gene therapy data
Fierce Biotech
Fri, 01/10/20 - 10:26 am
Ultragenyx
clinical trials
gene therapy
DTX301
Ultragenyx gene therapy data disappoints investors
Biopharma Dive
Thu, 09/5/19 - 08:15 pm
Ultragenyx
gene therapy
DTX401
glycogen storage disorder
clinical trials
Ultragenyx Inks a Partner-to-Buy Deal with GeneTx to Develop Treatment for Rare Genetic Syndrome
BioSpace
Thu, 08/15/19 - 12:20 pm
Ultragenyx
GeneTx Biotherapeutics
Angelman Syndrome
M&A

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