An investigation of the epidemiology and clinical characteristics of neuropathic pain in the UK Biobank has led to the discovery of a new pain gene and potential analgesic drug target in the peripheral nervous system. The gene, SLC45A4 (solute carrier 45A4), codes for a transporter that is involved in trafficking polyamines known to be involved in pain, across the cell membrane.

Duchenne muscular dystrophy is the most frequent inherited muscle disease and no cure is available. The condition is currently treated with corticosteroids, which can cause substantial side effects.

Inhibiting emopamil binding protein (EBP), an enzyme involved in cholesterol biosynthesis, promotes the formation of oligodendrocytes, a potential approach for the management of multiple sclerosis (MS); Genentech Inc. has presented data regarding their oral and brain-penetrant EBP inhibitor GNE-3406.

Loss-of-function mutations in the CTNNB1 gene cause β-catenin deficiency, leading to CTNNB1 syndrome, a rare neurodevelopmental disorder marked by motor and cognitive impairments. Because the disease stems from single-allele mutations that vary widely, a broadly applicable corrective strategy is needed. Since CTNNB1 is dosage-sensitive, therapies must preserve normal regulation, and in some cases may also need to suppress harmful mutant transcripts.

Jazz Pharmaceuticals plc and Saniona AB have entered into a global license agreement for Jazz to obtain exclusive worldwide rights to develop and commercialize SAN-2355 for epilepsy and other potential indications.