The FDA has approved Ctexli for the treatment of cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease, in ...
Lenacapavir, a twice yearly injection that prevents HIV transmission, was named the breakthrough medicine of 2024. But without US foreign aid dollars, its delivery to millions worldwide is under ...
Gain-of-function mutations in fibroblast growth factor receptor (FGFR) genes are known to cause a range of skeletal disorders ...