Myotonic dystrophy type 1 (DM1) is a rare, progressive genetic disease that causes severe muscle weakness and other debilitating symptoms, such as compromised respiration and cardiac conduction abnormalities. No disease-modifying therapy exists for DM1, so care focuses on managing symptoms like arrhythmia, myotonia, hypertension, cataracts, respiratory issues and sleep disorders.
Otsuka Pharmaceutical Co. Ltd. has identified new piperidine- and morpholine-carboxylate compounds acting as sortilin (SORT; NT3; Gp95) antagonists. As such, they are described as potentially useful for the treatment of amyotrophic lateral sclerosis, frontotemporal dementia, Alzheimer’s and Parkinson’s disease.
For treating bipolar disorder, investigators have explored single strategies targeting dopamine receptors or the glycogen synthase kinase-3 (GSK-3) pathway. Researchers at Istituto Italiano di Tecnologia and collaborators have used computational modeling and structure determination to improve on a prototype small molecule capable of simultaneously activating the dopamine D3 receptor while inhibiting GSK-3β.
Small fiber neuropathy (SFN) is a chronic and debilitating condition caused by damage to peripheral sensory nerves, which are responsible for pain, temperature sensation and autonomic functions. Current treatments are limited, highlighting the need for better therapies.
Cytokinetics Inc. has disclosed fast type myosin-binding protein C (MYBPC2) inhibitors reported to be useful for the treatment of neuromuscular disorders.
Congruence Therapeutics Inc. has closed a $32 million financing round to advance its pipeline of small-molecule correctors for diseases of protein misfolding.
Huntington’s disease is an inherited neurodegenerative disease in which the huntingtin protein undergoes polyglutamine expansion, leading to both loss and gain of functions. A research collaboration spanning France, Germany, the Netherlands and the U.S. showed in previous work that overexpressing CYP46A1 in the brain can mitigate disease in multiple mouse models.
Bright Minds Biosciences Inc. has released preclinical results for BMB‑201, a selective 5-HT2A/2C receptor agonist, in a validated isosorbide dinitrate (ISDN) rat model of vascular headache. BMB-201 is designed to harness the analgesic potential of serotonin modulation without the hallucinogenic effects commonly associated with 5-HT2A activation.
Humanwell Pharmaceutical US Inc. has described sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, arrhythmia, cough, urinary incontinence and multiple sclerosis.
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