An international working group has proposed a new research framework that stages Parkinson's disease (PD) and defines it based on the disease's underlying biology, rather than on clinical symptoms.
Accent Therapeutics Inc. has completed a $75 million series C financing to support progression of its lead programs through early clinical development.
The Cystic Fibrosis Foundation (CF Foundation) has agreed to provide Prime Medicine Inc. with up to $15 million to support the development of prime editors for the treatment of cystic fibrosis (CF).
Primary open-angle glaucoma is the leading cause of blindness globally. Individuals of African ancestry have a disproportionately high risk of developing glaucoma, but genetic risk factors have been studied mainly in populations of European ancestry.
Researchers from Maze Therapeutics Inc. presented the discovery and preclinical characterization of MZ-101 as a potential candidate for the treatment of Pompe disease and other glycogen storage disorders.
The discovery of how proteins fold and what happens if they don’t, or do it wrong, has impacted medicine. Protein function and several pathologies depend on folding. Four scientists, Franz-Ulrich Hartl, Arthur Horwich, Kazutoshi Mori and Peter Walter, revealed the role of chaperones in assisting proteins to acquire their correct structure and described a failure warning system, the unfolded protein response (UPR). For their studies, the researchers will receive the 2024 Frontiers of Knowledge Award in Biology and Medicine from the BBVA Foundation.
Mycosis fungoides (MF) is the most frequent form of cutaneous T-cell lymphoma. Although there are therapeutic options for the management of MF, long-term remission or complete cure are difficult to achieve with current drugs in the clinical setting.
China Pharmaceutical University has synthesized kaurane tetracyclic diterpenoid derivatives acting as NLRP3 inflammasome inhibitors reported to be useful for the treatment of arthritis and skin disorders.
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