资讯

BioSpace3 天
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 ...
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 ...
Olivia had been showing symptoms since she was just 7 months old — including a belly that remained swollen, hard to the touch ...
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 ...
Pharmabiz3 天
Ultragenyx receives CRL from US FDA for UX111 AAV gene therapy to treat Sanfilippo syndrome ...
Novato, California Monday, July 14, 2025, 16:00 Hrs [IST] ...
BioSpace1 天
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 ...
BioPharma Dive3 天
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 ...
The Pharma Letter3 天
Ultragenyx hit with FDA delay for gene therapy targeting rare childhood disorder
Ultragenyx Pharmaceutical (Nasdaq: RARE) has been dealt a regulatory setback, as the American medicines regulator has ...
Nature12 年
Disease correction by combined neonatal intracranial AAV and systemic ...
Mucopolysaccharidosis type IIIB (MPS IIIB) or Sanfilippo Syndrome type B is a lysosomal storage disease resulting from the deficiency of N-acetyl glucosaminidase (NAGLU) activity. We previously ...