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BioSpace4 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 ...
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 ...
Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat ...
Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are ...
Ultragenyx gets Complete Response Letter for Sanfilippo syndrome gene therapy
The U.S. FDA has issued a Complete Response Letter to Ultragenyx Pharmaceutical (NASDAQ:RARE) for its BLA for UX111 (ABO-102), its AAV gene therapy to treat Sanfilippo syndrome type A. The company ...
Pharmabiz4 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] ...
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, ...
Medical Marketing & Media1 小时
Rx Rundown: Sarep­ta Ther­a­peu­tics, Juul, Pfizer and more
Sino Biopharma will acquire LaNova Medicines, which has licensed cancer drugs for Merck and AstraZeneca, for $951 million.
Precision Medicine Online3 天
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.
The Pharma Letter4 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 ...
Investor's Business Daily on MSN3 天
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.
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 ...
BioWorld3 天
Ultragenyx hit again, this time with a CRL in rare disease
Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a ...