Liver glycogen storage disease type IX (GSD IX) accounts for 25% for all GSD cases, with a prevalence of 1 out of 100,000 patients. GSD IX is caused by deficiency in phosphorylase kinase (PhK), which is comprised of four subunits (α2, β, δ and γ2), with γ2 being the catalytic domain.
Research at China Hinye Pharmaceutical Co. Ltd. has led to the development of μ-opioid receptor agonists reported to be useful for the treatment of pain.
Galderma Holding SA has identified metabolites of CD-14547 acting as mammalian target of rapamycin (mTOR; FRAP1) inhibitors. As such, they are reported to be useful for the treatment of acne, atopic dermatitis, actinic keratosis and psoriasis.
Nitrogen-containing tricyclic compounds acting as pyruvate dehydrogenase kinase 2 (PDHK2; PDK2) inhibitors have been described in a recent Japan Tobacco Inc. patent. They are reported to be useful for the treatment of diabetes, atherosclerosis, cancer, chronic kidney disease, Alzheimer’s disease, heart failure, pulmonary hypertension and stroke.
Immunoprecise Antibodies Ltd.'s Talem Therapeutics LLC subsidiary has entered into a research collaboration and exclusive option license agreement with Xyphos Biosciences Inc., a wholly owned subsidiary of Astellas Pharma Inc.
Comanche Biopharma Corp. has received IND clearance from the FDA to conduct a first-in-human study of CBP-4888, its novel, small interfering ribonucleic acid (siRNA) therapy to treat pre-eclampsia.
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