Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the Best 52-Week Low Stocks to Buy According to Analysts. On July 14, ...

A discovery with major implications for cancer treatment has been made by a team of researchers from Rensselaer Polytechnic ...

It is trying to treat a devastating lifelong disease by fixing it at its source. We are able to fix the actual genetic ...

Fragile X disorders include two distinct conditions: fragile X syndrome (the most common single-gene cause of developmental delay and autism) and an entirely separate group of premutation syndromes.

RARE faces regulatory setback as FDA issues a CRL for UX111, delaying approval of the MPS IIIA gene therapy until 2026.

Ultragenyx's high burn rate, costly R&D, and recent setbacks increase dilution risk, impacting future profitability. Learn ...

Ultragenyx's UX111 gene therapy faces FDA delay due to facility issues; analysts adjust price targets amid uncertain ...

The firm said the agency cited chemistry, manufacturing, and controls issues in its complete response letter, but had no concerns about product quality.

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

Ultragenyx received a Complete Response Letter from the FDA for UX111 AAV gene therapy to treat Sanfilippo Syndrome Type A ...

The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...

Novato, California Monday, July 14, 2025, 16:00 Hrs [IST] ...