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

A new study led by Indiana University School of Medicine researchers revealed a potential strategy to address neurobehavioral challenges associated with neurofibromatosis type 1, or NF1, a genetic ...

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 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 ...