NS Pharma Inc.’s NS-051 (NCNP-04) has been awarded orphan drug designation by the FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping.
Genfit SA is discontinuing its VS-01 program in acute-on-chronic liver failure (ACLF), and has decided to reprioritize development of VS-01 for urea cycle disorder.
The FDA has cleared HDT Bio Corp.’s IND application for HDT-321, a first-in-class prophylactic designed to protect against Crimean-Congo hemorrhagic fever (CCHF).
Invasive fungal infections pose a significant global health challenge due to their severity and the scarcity of effective and safe treatment options. Unlike antibacterial drug development, creating new antifungals is especially challenging because fungal and human cells share a eukaryotic structure, highlighting the need for innovative treatment strategies.
Experts agree that the earlier Alzheimer's disease is detected, the sooner action can be taken. And so, the key to preventing deterioration is identifying the most effective early biomarkers, those that can spot the disorder and help halt its progression. Recent advances in the field have pushed a new era of early detection through blood-based biomarkers and personalized medicine strategies based on each patient’s genetic, immunological and clinical profile.
A new generative AI model trained on UK Biobank data can simultaneously forecast the risks and timing of developing over 1,000 different diseases a decade into the future. The developers applied similar algorithmic concepts to those used to develop large language models like ChatGPT and Gemini to build the model, using medical records from 402,799 participants in UK Biobank.
Touchstone Innovations Ltd. has described peptide hormone analogues acting as gastric inhibitory polypeptide receptor (GIPR) agonists reported to be useful for the treatment of obesity.
Hangzhou Zhongmei Huadong Pharmaceutical Co. Ltd. has divulged antibody-drug conjugates (ADCs) comprising antibodies targeting human mucin-17 (MUC17) covalently linked to a cytotoxic drug through a linker reported to be useful for the treatment of cancer.
Bionova Pharmaceuticals Ltd. has identified methylthioadenosine (MTA) synergistic protein arginine N-methyltransferase 5 (PRMT5) inhibitors reported to be useful for the treatment of cancer.
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