In previous work, researchers from Kawasaki Medical School and collaborating institutions engineered a modified HEXB construct, modHexB, to improve GM2 ganglioside (GM2) recognition and GM2-activating protein (GM2A) interaction. The team has now combined these previous advancements to develop a new gene therapy strategy for Sandhoff disease.
If Benjamin Braddock, of The Graduate fame, were a young neuroscientist in the 21st century instead of a liberal arts graduate in 1967, the advice he received from his parents’ neighbor might not have been “One word: plastics!” but “One word: plasticity!” Plasticity is a hot concept in neuropsychiatric disorders. New and old treatment modalities, these days, are said to work as psychoplastogens or neuroplastogens.
Sichuan Kelun Pharmaceutical Research Institute Co. Ltd. has identified G protein-coupled receptor 6 (GPR6) inverse agonists reported to be useful for the treatment of depression, bipolar disorder, substance abuse and dependence, eating disorders, schizophrenia, cognitive disorders, Parkinson’s disease and Huntington's disease, among others.
Chengdu Kanghong Pharmaceutical Group Co. Ltd. is developing a nonopioid Nav1.8 inhibitor, KHN-702. The effects of KHN-702 were evaluated both in vitro and in vivo, in a plantar incision pain model, and results were presented at the recent American Academy of Neurology meeting in Chicago.
Minimal residual disease (MRD) has become a central concept in modern oncology, reshaping how clinicians evaluate response, relapse risk and treatment precision. As increasingly sensitive technologies reveal traces of cancer that persist after therapy, MRD is emerging as both a biological challenge and a clinical opportunity, especially as new data illuminate its complexity across hematologic and solid tumors. This topic was addressed at the 2026 American Association for Cancer Research (AACR) annual meeting.
Chengdu Mfs Pharma Co. Ltd. has prepared and tested compounds for the potential treatment of pain, sleep disorder and status epilepticus epilepsy as well as for use as an anesthetic.
At the recently concluded meeting of the American Academy of Neurology, Synendos Therapeutics AG reported preclinical data from the pharmacological characterization of SYT-510, which is the first selective endocannabinoid reuptake inhibitor (SERI) to enter the clinic for the potential treatment of disorders affecting the CNS, such as anxiety or movement disorders. A new class of endocannabinoid system (ECS) modulators, SERIs potently and selectively inhibit endocannabinoid reuptake to help the ECS restore normal brain function under disease conditions.
Singularity Pharma Ltd. has divulged mitragynine analogues that are potentially useful for the treatment of postoperative pain, depression, opioid dependence, opioid withdrawal, inflammatory and psychiatric disorders.
Entos Pharmaceuticals Inc. has established a collaboration with the L-CMD Research Foundation with the aim of developing a curative therapy for LMNA-related congenital muscular dystrophy (L-CMD).
Researchers from Xenon Pharmaceuticals Inc. described the preclinical efficacy of XPC-837 in models of Dravet syndrome, a severe developmental and epileptic encephalopathy most commonly caused by de novo loss of function mutations in the SCN1A gene.
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