Autifony Therapeutics Ltd. has described compounds acting as modulators of voltage-gated potassium channels from the Kv3 subfamily, such as Kv3.1, Kv3.2 and/or Kv3.3, reported to be useful for the treatment of myoclonic epilepsy, among others.

The FDA has awarded orphan drug designation...

Bacteria inflaming the meninges have developed an immunosuppressive mechanism that contributes to their ability to attack the brain. Researchers found that, by activating pain receptors (nociceptors) to release chemical substances that block an immune cell receptor, Streptococcus pneumoniae and Streptococcus agalactiae deactivated the protective function of macrophages and weakened brain defenses. This, in turn, enabled them to invade the brain.

A recent Janssen Pharmaceutica NV patent describes monoglyceride lipase (MGLL...

Treatment with a cell-penetrating peptide that prevented nuclear export of unprocessed C9ORF72 RNA and its subsequent translation into neurotoxic dipeptide repeat proteins reduced motor neuron damage and death both in fruit fly models of amyotrophic lateral sclerosis (ALS), and in patient-derived induced neuronal precursor cells (iNPCs). The work suggests that targeting nuclear export could be a therapeutic option in ALS, and possibly also frontotemporal dementia (FTD), where C9ORF72 mutations also play a role.