News

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 ...
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 ...
Zacks Investment Research on MSN3d
Ultragenyx Pharmaceuticals Gets CRL for UX111 Gene Therapy for MPS IIIA
Ultragenyx Pharmaceuticals RARE announced that the FDA has issued a Complete Response Letter (CRL) for its biologics license ...
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 ...
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 ...
Investor's Business Daily on MSN4d
Ultragenyx Pharma Takes Another Hit As FDA Slaps Its Gene Therapy With A Rejection
Ultragenyx Pharmaceuticals took another hit when the FDA rejected its gene therapy for patients with a rare, genetic disorder.
BioPharma Dive4d
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 ...
Pharmabiz4d
Ultragenyx receives CRL from US FDA for UX111 AAV gene therapy to treat Sanfilippo syndrome type A
Novato, California Monday, July 14, 2025, 16:00 Hrs [IST] ...
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 ...
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, ...