Accumulation of fat molecules is detrimental to the cell. Researchers have made a breakthrough in understanding how our cells manage to stay healthy by recycling important fat molecules. Their study ...
Breakthrough NUS discovery sheds light on cellular recycling mechanism and potential for treating rare diseases Singapore ...
Gideon Bennett has a rare genetic disorder that makes even a small amount of protein dangerous, but the special medical food ...
Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or inactivating genes. Precisely fixing disease-causing mutations is far more ...
In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed ...
One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...
By Julie Parr Click here for updates on this story TEXARKANA, Texas (KTBS) — A Texarkana, Texas boy battling a rare genetic ...
Researchers at Washington University School of Medicine in St. Louis have conducted a longitudinal study on an individual ...
Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...
Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder ...
A surprising new study suggests that COVID-19 may not have originated from bats or pangolins, but rather from a rare fusion of human diseases. Using an advanced AI-driven approach called max-logistic ...