As patients with Pompe disease age, they develop neurocognitive issues and impairment in their cerebral white matter, as shown in MRI scanning. To date, there are no biomarkers that reflect this disease progression in the brain. The usefulness of neurofilament light chain (NfL) was investigated for this purpose.
Glutaric acidemia type I (GA1) is caused by severe deficiency in enzyme glutaryl-CoA dehydrogenase activity, which results in an impairment on lysine catabolism. These patients have accumulation of glutaric acid and glutaryl carnitine, with loss of striatal neurons and atrophy of the frontotemporal cortex. The aim of investigators was to investigate plasma markers of neurodegeneration and inflammation, such as brain-derived neurotrophic factor (BDNF) and cathepsin D in patients (N = 6) with GA1 and matched healthy controls.
Researchers from Istituto di Ricerche Farmacologiche Mario Negri aimed to characterize lectin pathway (LP) activation after ischemic stroke in a novel humanized mouse model expressing human mannose-binding lectin.
Treatment with injections of gonadotropin-releasing hormone (GnRH), indicated to prevent sexual maturation deficits in Down syndrome, also reduced cognitive function impairment associated with Down syndrome, also called trisomy 21. With age, about three-quarters of people with Down syndrome develop Alzheimer's disease. They also lose their sense of smell. Both circumstances could improve with pulse doses of GnRH, according to a study led by the French National Institute of Health and Medical Research (Inserm) and the University of Lausanne (UNIL) published in the Sept. 1, 2022, issue of Science.
Researchers from Pfizer Inc. presented preclinical data for the melanocortin MC4 receptor (MC4R) antagonist PF-07258669, being developed for the treatment of anorexia nervosa.
Circadian rhythm disorders are associated with psychiatric disorders including depression, bipolar disease and schizophrenia. To target casein kinase I isoform delta (CKI-delta), Janssen Pharmaceutica NV scientists sought potent, selective, brain-penetrant CKI-delta inhibitors which can be dosed daily and orally.
Biodol Therapeutics, a portfolio company of V-Bio Ventures, has nominated its first preclinical candidate for neuropathic pain. The candidate is a small-molecule allosteric FLT3 inhibitor, suitable for chronic oral administration and without the safety limitations of less specific intracellular kinase inhibitors.
Target ALS Foundation Inc. and the Alzheimer's Drug Discovery Foundation LLC (ADDF) have announced the first four award recipients in a new initiative to identify and develop biomarkers for neurodegenerative diseases, including Alzheimer's disease, amyotrophic lateral sclerosis (ALS) and frontotemporal degeneration (FTD).
Trigeminal neuralgia is a debilitating chronic neuropathy characterized by sudden, short and intense episodes of shooting, stabbing or shock-like pain in the face. To date, there is still an incomplete understanding of the pathophysiologic molecular mechanisms behind trigeminal neuralgia and the search for molecular targets is of medical need.
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