A loss of BAG3 leads to the accumulation of misfolded and damaged proteins, impairing the heart’s ability to contract. Rocket ...

The Centers for Medicare and Medicaid Services (CMS) has selected 35 participants for its new, voluntary Cell and Gene ...

Ultragenyx Pharmaceuticals RARE announced that the FDA has issued a Complete Response Letter (CRL) for its biologics license ...

- ZEVASKYN is the only FDA-approved therapy to treat RDEB wounds with a single application - ...

This is the first time such results have been achieved in both children and adult patients born with a specific type of ...

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

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...

The OUTREACH Study – Demonstrating that CAR-T therapies can be effectively administered in community cancer settings: In the ...

Alfie Clatworthy and Angus Powell are two of only three children in the UK living with CRELD1 and there is currently no cure.

IT之家 7 月 4 日消息，7 月 2 日，东南大学附属中大医院耳鼻咽喉头颈外科柴人杰教授，联合多家单位在国际顶级医学期刊 Nature Medicine 发表题为《AAV gene therapy for autosomal recessive ...

In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy ...

A single shot of gene therapy restored hearing in people born deaf, with most showing improvement within weeks and some ...