Mutations in the KCNT1 gene produce gain-of-function effects that lead to overactivation of the potassium channel and consequent disruption of normal neuronal electrical signaling. These alterations give rise to a severe, early-onset developmental and epileptic encephalopathy that is typically associated with a high seizure burden and resistance to standard antiseizure medications.
In an alternative to small molecules, researchers at the University of Ferrara and Università degli Studi di Padova sought to create an NOP agonist based on peptides, specifically dimers of peptides derived from native nociceptin.
Epiminder Ltd. raised AU$125 million (US$82.99 million) in its initial public offering on the Australian Securities Exchange to commercialize its Minder system, a long-term ambulatory electroencephalography monitoring device for epilepsy.
People with drug-resistant epilepsy have had few other options, but Neuropace Inc. appears on target to provide an alternative. The Nautilus trial of its responsive neurostimulation system for individuals with drug-resistant idiopathic generalized epilepsy showed a 77% reduction in generalized tonic-clonic seizures sustained over 18 months.
Positive efficacy results led to Praxis Precision Medicines Inc.’s phase II Embold study in developmental and epileptic encephalopathies (DEEs) being halted early, propelling the company’s shares dramatically upward.
The ALS Association has announced the recipients of its 2025 Hoffman ALS Clinical Trial Awards. These research grants, worth up to $1 million each, support early-phase trials that generate critical data to accelerate the development of new amyotrophic lateral sclerosis (ALS) treatments.
Drug developers big and small are pursuing a solution for the curse of psychosis that occurs along with the already tragic diagnosis of Alzheimer’s disease, an affliction known as ADP.
A little over a week after announcing that the Evoke and Evoke+ studies failed to show that oral semaglutide could slow cognition decline in patients with Alzheimer’s disease, investors and researchers got the first look at the actual data from the studies, which were presented at the Clinical Trials on Alzheimer’s Disease 2025 meeting.
The U.S. FDA granted accelerated approval for the Biologics License Application for Axogen Inc.'s Avance, an acellular nerve allograft that serves as a scaffold for nerve regrowth, for treatment of patients with sensory, mixed and motor peripheral nerve discontinuities. The company expects Avance to be commercially available in the second quarter of 2026.
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