Vitamin D is not only an essential nutrient, but also the precursor of the hormone calcitriol, indispensable for health. It ...

Mantle cell lymphoma treatment offers options if patients relapse after CAR T-cell therapy, from bispecific antibodies, antibody drug conjugates, and other emerging therapies.

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 ...

The National Cancer Center Research Institute in Tokyo reports that a newly isolated gut bacterium, designated ...

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 Pharmaceuticals took another hit when the FDA rejected its gene therapy for patients with a rare, genetic disorder.

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 ...