While glucocorticoids are used as first-line therapy for Duchenne muscular dystrophy (DMD), these agents are associated with several adverse events, including accelerated bone loss and muscle atrophy. As a result, prolonged glucocorticoid treatment is one of the primary contributors to the high fracture rate in patients with DMD.

For most psychiatric illnesses, the precipitating event is mysterious. Many conditions are thought to result from a mix of genetic risk and environmental factors, but the specific trigger remains unknown. In post-traumatic stress disorder (PTSD), the environmental trigger is usually clear. In many cases, it is all the affected individuals can think about. “Intrusive reliving” of the triggering situation is one of the core features of PTSD.

Küleon LLC (formerly Psilosterics LLC) has announced a trifunctional serotonergic small molecule that is a full 5-HT2C receptor agonist and full antagonist of the 5-HT2A and 5-HT2B receptors with potential to treat neuropsychiatric disorders. Designated KB-128, it could be developed to treat disorders that can be modulated though 5-HT2C receptors, including schizophrenia, Alzheimer’s psychosis, depression, obesity and addiction.

Ionis Pharmaceuticals Inc. has announced its intention to advance four new wholly owned neurology medicines into the clinic by the end of next year.

The broadest view of post-mortem brains in Alzheimer’s disease (AD) has unveiled the genome, transcriptome and epigenome alterations of this neurodegenerative condition. The coordinated research, directed by scientists at the Massachusetts Institute of Technology (MIT), also described new cellular pathways that could help the scientific community design new therapies. Four simultaneous studies published on Sept. 28, 2023, in Cell, presented a brain single-cell atlas of AD, exposed the damage that affects DNA, and described the processes that alter the microglia and dysregulate the epigenome.

Oryzon Genomics SA has announced two grants that will support the exploration of the role of epigenetic targets in the treatment of neuronal pathologies. Funded by the Spanish State Research Agency and the Ministry of Science and Innovation, the grants cover two public-private collaboration projects.