Browse diseases

…flects a strong commitment to improving care and visibility for rare diseases, while highlighting the financial challenges of ODs in small he…

…d observations. This is particularly relevant in the context of rare diseases, where real-world data are often fragmented, siloed or insuffic…

…r diseases, exemplified by small interfering RNA treatments for hereditary transthyretin amyloidosis, which use liver-targeting strategies such as GalNAc conjugatio…

…omises to improve the health and well-being of individuals with rare diseases1 2 and describes an approach to disease prevention and treatment…

… vision and generative models are enabling earlier detection of rare diseases, dynamic risk stratification and personalised care pathways. Em…

…rce allocation decisions for high-cost drugs for rare diseases (DRDs) raise several challenges for decision makers, and, given the c…

…nse oligonucleotide (ASO) therapies have dominated approvals in rare diseases. Many patients with a rare genetic disease stand to benefit fro…

…ng an LLM-based Agentic system, it can handle information about rare diseases. Our proposed Agentic system uses an open-source LLM, named Med…

…Rare diseases affect an estimated 350 million people worldwide, over 80% of w…

…Worldwide, approximately 80% of rare diseases are thought to have an inherited component. Of these, around 75…

…rapeutic platforms have brought promising solutions for various neurodegenerative/neuromuscular disorders and rare diseases. Currently, 20 oligonucleotide drug products …

…sting for surgical devices. Its applications include simulating rare diseases, modelling diverse patient demographics, and generating early e…