Despite being known for more than 150 years, Duchenne muscular dystrophy (DMD) remains an untreatable disease affecting approximately 1 of every 3,500-5,000 males. Muscles in patients express no or inactive dystrophin, rendering them weak and less mobile.
Nipah virus, which circulates in fruit bats and other animals primarily in Asia and the South Pacific, causes severe neurological respiratory disease in humans and leads to death in 40%-75% of cases. The virus is endemic in Bangladesh and India, where outbreaks have occurred nearly annually since 2001.
The specific tau isoforms, such as 3-repeat (3R) and 4-repeat (4R) isoforms, and the distinct conformational strains that misfolded tau can adopt are determinants of the molecular and clinical heterogeneity observed across tauopathies.
CSPC Pharmaceutical Group Ltd. has obtained clinical trial approval from China’s National Medical Products Administration (NMPA) for SYH-2046 for heart failure after acute myocardial infarction.
Cutaneous melanoma nearly always arises on parts of the body that receive abundant sun but, rarely, it can arise on parts that do not, such as the palms of the hands or soles of the feet. These rare cases of acral and mucosal melanomas, which often feature mutations in the transmembrane tyrosine kinase KIT, do not respond to current melanoma therapies.
Researchers have developed a new compound that can prevent long COVID symptoms in mice that could lead to a future drug for the debilitating condition in humans. Developed by researchers at the Walter and Eliza Hall Institute of Medical Research (WEHI) in Melbourne, the world-first study found mice treated with the antiviral compound were protected from long-term brain and lung dysfunction, which are key symptoms of long COVID.
Scientists at Zai Lab (Shanghai) Co. Ltd. and Zai Lab (US) LLC have described poly(ADP-ribose) glycohydrolase (PARG) inhibitors reported to be useful for the treatment of cancer.
Shanghai Jemincare Pharmaceuticals Co. Ltd. has identified androgen receptor antagonists reported to be useful for the treatment of cancer, alopecia, acne, hirsutism, polycystic ovary syndrome, precocious puberty, spinal and bulbar muscle atrophy, and age-related macular degeneration.
Oxford University Innovations Ltd. has synthesized hypoxia-activated proteolysis targeting chimeras (hypoxia-activated PROTACs; HAP-TAC) comprising a hypoxia-activated moiety modified E3 ubiquitin ligase-binding moiety coupled to a protein targeting moiety through a linker reported to be useful for the treatment of cancer.
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