ETHYPHARM

Early trials of antisense oligonucleotide (ASO) treatments for rare genetic epilepsies show promise in reducing seizures, but high costs pose challenges. ASOs have grown from a single N-of-1 study in 2019 to involve over two dozen patients, with some trials reporting significant seizure reductions. Despite promising results, the high cost of ASO treatments, potentially in the millions, raises concerns about accessibility. Neurologists emphasize the need for genetic screening at birth to identify patients early and streamline the development of these gene-based therapies.

Prescribing medical cannabis for chronic pain on the NHS could save £4 billion annually, improve health outcomes, and boost the UK economy by £5.45 billion, according to a study by the Cannabis Industry Council and Drug Science.

K.W. is a member of the ACTIVE Workgroup supported by Alkermes, Dicerna, Ethypharm, Lundbeck, Mitsubishi, and Otsuka. J.L.M., B.P.L., and M.C.M. are consultants for GlaxoSmithKline, Novo Nordisk, HepaTX, and Prodigy. M.C.M. also serves on the Board of Amygdala Neuroscience and receives research support from Durect. Other authors declare no competing interests.