News

Bank of America Securities Reiterated a Buy Rating on Ultragenyx Pharmaceutical (RARE)
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the Best 52-Week Low Stocks to Buy According to Analysts. On July 14, ...
William Blair Maintains a Buy on Ultragenyx Pharmaceutical (RARE)
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the best oversold NASDAQ stocks to buy now. On July 14, William Blair ...
Her Belly Was Hard, Her Diapers Always Full. Why Her Mom Knew Something Was Wrong, Despite Doctors' Dismissals (Exclusive)
Olivia had been showing symptoms since she was just 7 months old — including a belly that remained swollen, hard to the touch ...
BioPharma Dive3d
FDA rejects Ultragenyx gene therapy over manufacturing concerns
While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...
BioSpace4d
Ultragenyx Takes Second Hit as FDA Rejects Gene Therapy for Sanfilippo Syndrome
The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...
US FDA declines to approve Ultragenyx's gene therapy for rare inherited disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
BioSpace2d
FDA Layoffs, 200 Rejection Letters, User Fees and Priority Vouchers, Bad Week for Rare Disease
Around 3,500 FDA employees received termination emails; FDA Commissioner Marty Makary suggests lowering industry user fees ...
GlobalData on MSN4d
Ultragenyx receives complete response letter for UX111 BLA
The FDA's request stems from observations made during recent inspections of Ultragenyx's manufacturing facilities.
Ultragenyx Pharmaceutical, Apple And Other Big Stocks Moving Lower In Monday's Pre-Market Session
Ultragenyx received a Complete Response Letter from the FDA for UX111 AAV gene therapy to treat Sanfilippo Syndrome Type A ...
Ultragenyx: The High Stakes Of Binary Events And A Short Cash Runway
Ultragenyx's high burn rate, costly R&D, and recent setbacks increase dilution risk, impacting future profitability. Learn ...
HuffPost UK on MSN18d
'She Stopped Talking': 5-Year-Old’s Dementia Diagnosis Began With These Early Warning Signs
The Sanfilippo Children’s Foundation notes: “As the brain gets progressively damaged, children experience severe hyperactivity, disordered sleep, loss of speech, cognitive decline, cardiac issues, ...
US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...