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, ...
Zacks Investment Research on MSN2d
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 ...
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 ...
BioSpace3d
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 ...
BioSpace1d
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 ...
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 ...
GlobalData on MSN3d
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 ...
Pella brings coloring book to life while also honoring community members
Animal statues can be seen across downtown Pella, bringing a children’s story to life while also honoring special community ...
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 ...
Pharmabiz3d
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] ...
Precision Medicine Online3d
FDA Declines to Approve Ultragenyx's Gene Therapy for Sanfilippo Syndrome Type A
The firm said the agency cited chemistry, manufacturing, and controls issues in its complete response letter, but had no concerns about product quality.