Pompe disease is a disorder caused by deficiency of the lysosomal acid α-glucosidase (GAA) enzyme, which leads to the accumulation of glycogen within the lysosomes, overall in skeletal and cardiac muscle.
Because selective BET inhibitors present better efficacy and safety than pan-BET inhibitors, current research is focused on the development of BD1- or BD2-selective inhibitors.
Almirall S.A. has entered into an exclusive license agreement with Novo Nordisk A/S for global rights to develop and commercialize the IL-21-blocking monoclonal antibody NN-8828 in certain fields, including immune inflammatory dermatological diseases.
The company has developed a method of transplantation of human hematopoietic stem cells that allows their engraftment and differentiation into microglial cells.
Researchers at the University of Ottawa have reported on the use of vanoxerine as a suppressor of CSC tumorigenic activity in preclinical models of colorectal cancer.
Vidac Pharma Holdings plc has released promising results for VDA-1275 in mouse cancer and human cellular organoid models of solid tumors, with VDA-1275 showing efficacy as a monotherapy and synergistic effects in combination with sorafenib and cisplatin.
In a recent study, researchers from Technische Universität Dresden and affiliated organizations evaluated the novel small-molecule inhibitor of the CBP/p300 HAT domain, A-485, as a potential therapeutic candidate for the treatment of hematological cancers and other pathologies related to blood leukocyte compartment.
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