Dyne Therapeutics Inc. is advancing novel therapeutics for people living with genetically driven neuromuscular diseases. Representatives from the company recently presented a poster showcasing preclinical and early clinical results from the development of their proprietary FORCE platform applied to Duchenne muscular dystrophy (DMD).
Ono Pharmaceutical Co. Ltd. has entered into a drug discovery collaboration agreement with Reborna Biosciences Inc. to generate RNA-targeting novel small molecules in the field of the central nervous system.
In the brain, molecular information is transmitted between cells through neural circuits. Synapses establish connections between the pathways that run from one area to another, allowing the most complex organ in the body to fulfill different functions. Cells and neural circuits are the basic biological elements in the study of mental illness. However, the scientific community still does not know how to interpret their role in neuropsychiatric disorders.
The EMA has rejected the Alzheimer’s disease therapy Kisunla (donanemab) from Eli Lilly and Co., saying the benefits of the anti-amyloid antibody do not outweigh the risks of edema and hemorrhage in the brain, known as amyloid-related imaging abnormalities (ARIA).
Researchers from F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have presented NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of neurodegeneration.
Previous studies have shown that protein expression of DOCK7 is increased in skeletal muscle biopsies from patients with Duchenne muscular dystrophy (DMD), leading researchers from the University of Alabama at Birmingham and affiliated organizations to assess the functional impact of DOCK7 on normal muscle and embryonic development of zebrafish.
Alzamend Neuro Inc. recently presented pharmacokinetic data for the lithium-based sparing therapy AL-001, a lithium salicylate/L-proline co-crystal, in 5XFAD mice, a murine model of Alzheimer’s disease (AD).
Researchers from the University of Queensland recently provided details on the discovery and preclinical characterization of a new hematopoietic prostaglandin D2 synthase (HPGDS) inhibitor, CLS-189, being developed for the treatment of Duchenne muscular dystrophy (DMD).
A variant burden analysis for bipolar disorder was performed using gene-based aggregation of loss-of-function variants in whole-genome sequencing data from Iceland and the UK Biobank; the association between bipolar disorder and the burden of loss-of-function variants was tested in 13,786 genes.
The first disease modifying therapies for Alzheimer’s may have limited utility in some senses, but they will be a force for change, providing momentum and altering the way governments as payers, and health systems as carers, think about the disease.
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