This article provides an end-to-end compendial framework to understand why compliance with pharmacopoeia standards is challenging.
FDA Approves First Treatment for Rare Lipid Storage Disease Cerebrotendinous Xanthomatosis
February 24th 2025Cerebrotendinous xanthomatosis, or CTX, is a metabolic disorder caused by a gene mutation that results in a deficiency of an important enzyme in the ability of the human body to break down fats.
FDA Grants Full Approval for Symptomatic Tenosynovial Giant Cell Tumor Treatment
February 18th 2025Tenosynovial giant cell tumor (TGCT) is rare and non-malignant, forming in or near joints, but which if left untreated can cause damage to joints and surrounding tissue, potentially leading to significant disability.