1+ week, 5+ day ago   (384+ words) Why does fat accumulation lead to serious liver damage in some people but not others? New research points to a cellular pathway that may help determine when metabolic stress progresses to inflammation and fibrosis—and suggests a way to intervene…...

2+ week, 4+ day ago   (1169+ words) SonoThera's founder and CEO says ultrasound could overcome genetic medicine's biggest delivery challenges, including payload size, tissue targeting, and repeat dosing By Jonathan D. Grinstein, PhD The potential is enormous because targeting is driven largely by physics rather than a biological…...

3+ week, 2+ day ago   (225+ words) Rare disease specialist Ultragenyx’s gene therapy Genglycos has received accelerated approval from the FDA for treatment of glycogen storage disease type Ia (GSDIa), a rare metabolic condition. This marks the fifth FDA approval for the California-based biotech, which has already…...

3+ week, 3+ day ago   (401+ words) Mayo Clinic researchers have quantified the risk that pancreatic cysts will progress to pancreatic cancer based on the number of worrisome features they display. Published in Gastroenterology, the findings could help doctors better assess which cysts are most likely to…...

3+ week, 5+ day ago   (481+ words) Researchers in Australia have developed a gene therapy that could restore heart function in children born with mutations that cause cardiomyopathy. A study published in Nature Cardiovascular Research suggests this approach could potentially address more than 25% of cardiomyopathies that have…...