Limb-girdle muscular dystrophy type 2D (LGMD2D/R3) is a rare genetic disorder caused by mutations in the SGCA gene, leading to defective folding and the loss of functional α-sarcoglycan, with progressive muscle degeneration. There is currently no approved treatment targeting the underlying cause of the disease.
Melanoma is the most aggressive form of skin cancer and often spreads to the brain. Though immunotherapy has greatly improved the outlook for even metastatic melanoma patients, once melanoma brain metastases (MBM) develop, prognosis worsens, and available therapeutic options decline. Scientists at the Institute for Neurosciences (IN), a joint center of the Spanish National Research Council (CSIC) and the Miguel Hernández University of Elche (UMH) have found a way to tackle MBM through microglial reprogramming.
Curently the only available blood test to tell which women are at risk of postpartum depression (PPD) is the one that confirms their pregnancy – and a one in seven chance of developing the condition which can have long-term implications for both mother and child. Research out of the University of Virginia and Weill Cornell Medicine could soon change that by identifying biomarkers in the third trimester of pregnancy that indicate which women have the greatest risk of developing PPD.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have described NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of neurodegeneration.
Redx Pharma plc has divulged Rho kinase 1 (ROCK1) and 2 (ROCK2) inhibitors reported to be useful for the treatment of autoimmune disease, cancer, fibrosis, inflammatory and neurological disorders.
Merck Sharp & Dohme LLC has identified targeted activator of cell kill (TACK) compounds acting as Gag polyprotein (HIV-1)/protein Pol dimerization inducers reported to be useful for the treatment of HIV infection.
Shanghai Yidi Biotechnology Co. Ltd. has synthesized proteolysis targeting chimeras (PROTACs) comprising an E3 ubiquitin ligase coupled to an androgen receptor targeting moiety via a linker reported to be useful for the treatment of spinal and bulbar muscular atrophy, prostate and breast cancer.
Shenzhen Zhongge Biotechnology Co. Ltd. has disclosed tyrosine-protein phosphatase non-receptor type 2 (PTPN2; TCPTP) inhibitors reported to be useful for the treatment of cancer.
Oncolytic viruses are therapeutic agents used for in situ immunization in cancer immunotherapy. Unfortunately, its efficacy is particularly limited in solid tumors expressing stimulator of interferon genes (STING) and retinoic acid-inducible gene I (RIG-I).
Triple-negative breast cancer (TNBC) is one of the most aggressive and difficult-to-treat forms of breast cancer, lacking targeted therapies due to its molecular characteristics. Tumor-associated neutrophils (TANs), particularly the pro-tumor N2-type, contribute to TNBC progression and resistance to treatment.
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