Fragile X syndrome (FXS) is the most common inherited form of intellectual disability and a leading monogenic cause of autism, yet effective treatments remain elusive. Previous work showed that N-methyl-D-aspartate receptors (NMDARs) play a prominent pathophysiological role in FXS and other neurodevelopmental disorders.

Researchers from Medical University of Vienna and affiliated organizations have presented findings from a study that aimed to assess the perilymph and tissue distribution of AC-102, a small and lipophilic intratympanically delivered pyridoindole derivative, in clinical development at Audiocure Pharma GmbH for the treatment of idiopathic sudden sensorineural hearing loss.

Tyrosine kinase 2 (TYK2), expressed in astrocytes and microglia, is involved in the activation of pathways triggered by proinflammatory cytokines, such as IL-23, IL-12 and type I interferons (IFNs), within the central nervous system (CNS). Dysregulated activation of astrocytes and microglia may contribute to the neuroinflammation associated with progressive forms of multiple sclerosis (MS).

Guillain-Barré syndrome (GBS) is an immune-driven inflammatory disorder of the peripheral nervous system characterized by muscle weakness and paralysis. Despite treatment options, GBS stays severe, with a mortality rate of 3%-10%. The mechanisms behind GBS are poorly understood and new therapeutic options are needed.

Aminoglycoside antibiotics are essential for treating some severe bacterial infections but are notorious for causing irreversible hearing loss in 20%-47% of patients.