RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.

March 12 (Reuters) - Ultragenyx Pharmaceutical said on Thursday its experimental gene therapy helped patients in a late-stage trial to better control ammonia levels linked to a rare inherited disorder ...

Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...

Thanks to advances in imaging and diagnostic technologies, clinicians can now detect many genetic disorders in the womb, ...

Researchers have used gene therapy to partially reprogram the brain cells that store memories in aging mice, reversing signs of cellular decline and restoring learning ability. The technique, ...

Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid ...