A large-scale study cross-referencing genomic data from multiple sources with primary care health records has identified genetic overlaps in 72 chronic diseases, opening the way for a more holistic approach to researching, treating and preventing multimorbidity. Read More

Fabry disease is an X-linked lysosomal storage disorder caused by a deficiency of the enzyme α-galactosidase A (GLA), which leads to complex multisystemic involvement, with the majority of patients developing hypertrophic cardiomyopathy. Read More

Bioray Pharmaceutical Co. Ltd. has announced clinical trial clearance in China by the National Medical Products Administration (NMPA) for BR-111 for injection for the treatment of ROR1-positive hematological malignancies and solid tumors. Read More

Researchers at E-Therapeutics plc recently presented efficacy and safety data on ETX-148, a pan-hemophilia agent in murine models of hemophilia A and B. Read More

At the recent annual congress of the European Association for Haemophilia and Allied Disorders, researchers from Novalgen Ltd. presented the discovery and preclinical characterization NVG-444, a potentially first-in-class next-generation FVIII mimetic antibody equipped with autoregulation to reduce prothrombotic risks that is being developed for the treatment of hemophilia A. Read More

With the first company in the world announcing more than $1 billion in annual revenue from pulsed field ablation on Feb. 5, Clarivate plc and BioWorld MedTech’s latest report provides well-timed insight into the stunning growth and bright future of this new medical technology for the treatment of atrial fibrillation. Read More

Researchers from Huazhong University of Science and Technology have described the potential use of RBM47 as a therapeutic target for thyroid-associated ophthalmopathy, an autoimmune disorder characterized by proptosis, lid swelling, diplopia and compressive optic neuropathy. Read More

Researchers from Fudan University and Southwest University published data from a study that detailed the development of a novel vertebrate model suitable for high-throughput screening of potential antiaging compounds. Read More

Tuojie Biotech (Shanghai) Co. Ltd. has prepared oxygen-containing fused tricyclic derivatives acting as CDK2/cyclin E1 and/or CDK4/cyclin D1 inhibitors. As such, they are reported to be potentially useful for the treatment of cancer, infections, autoimmune disease and inflammatory disorders. Read More

Work at Greenstone Biosciences Inc. has led to the identification of dihydroartemisinin derivatives reported to be useful for the treatment of fibrosis. Read More

CSPC Pharmaceutical Group Ltd. has gained clinical trial clearance from China’s National Medical Products Administration (NMPA) for SYS-6017, an mRNA vaccine to prevent herpes zoster infections. Read More

The Brigham and Women's Hospital Inc., Massachusetts General Hospital and UCL Business Ltd. have jointly patented new benzo[c][l,2,5]thiadiazolyl compounds and radiolabeled derivatives targeting α-synuclein, amyloid-β (Aβ) protein and microtubule-associated protein tau. Read More

Enanta Pharmaceuticals Inc. has discovered mast/stem cell growth factor receptor kit (KIT; c-KIT; CD117) inhibitors reported to be useful for the treatment of cancer, autoimmune disease, fibrosis, pulmonary arterial hypertension, irritable bowel syndrome, inflammatory, dermatological and respiratory disorders, among others. Read More

Fanconi anemia (FA) is a rare genetic disorder characterized by defective DNA repair, leading to hematopoietic stem cell (HSC) dysfunction and bone marrow failure. Replication stress, which compromises HSC maintenance and regeneration, is one of the central underlying mechanisms in FA. Read More

Muna Therapeutics ApS has disclosed new potassium voltage-gated channel subfamily A member 3 (KCNA3, Kv1.3) channel blockers reported to be useful for the treatment of cancer, neurodegeneration, cardiovascular, gastrointestinal, immunological, inflammatory, metabolic and renal disorders, among others. Read More