Dravet syndrome is a rare, severe, lifelong developmental and epileptic encephalopathy that begins in infancy and is marked by prolonged, often fever-triggered seizures that are difficult to control. It is usually caused by mutations in the SCN1A gene and is associated with developmental delay, cognitive and behavioral impairment, and reduced life expectancy.
Researchers from Xenon Pharmaceuticals Inc. described the preclinical efficacy of XPC-837 in models of Dravet syndrome, a severe developmental and epileptic encephalopathy most commonly caused by de novo loss of function mutations in the SCN1A gene.
Stoke Therapeutics Inc.’s speeded-up timeline for zorevunersen, the antisense oligonucleotide in development with Biogen Inc. as a first-in-class potential disease-modifying treatment for Dravet syndrome, put the rare, severe form of lifelong epilepsy in the spotlight. The news involved completion of enrollment and a phase III data readout from the Emperor study, as officials said signups of 150 patients are expected in the second quarter of the year, which puts the study on track for data in mid-2027.
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.
Dravet syndrome (DS) is a rare and severe form of epilepsy that causes intellectual disability and motor deficits and can lead to premature death. A loss-of-function mutation in one copy of SCN1A, encoding the voltage-gated sodium channel (NaV)1.1 α subunit, is the most frequent alteration found in DS patients and has been linked with inhibitory neuron dysfunction. Despite the potential of gene therapies, AAV-mediated SCN1A gene replacement for DS has not been possible yet due to AAV genome size constraints.
Selene Therapeutics Ltd. has identified brain permeable dihydroorotate dehydrogenase (DHODH) inhibitors reported to be useful for the treatment of epilepsy, cancer, Alzheimer’s disease, rheumatoid arthritis and multiple sclerosis.
With a move into Lilly Gateway Labs in Boston’s Seaport District, privately held Tevard Biosciences Inc. is ramping up development of its transfer RNA (tRNA)-based therapies to cure everything from Dravet syndrome and other neurological conditions to cardiology indications and muscular dystrophies.
With a move into Lilly Gateway Labs in Boston’s Seaport District, privately held Tevard Biosciences Inc. is ramping up development of its transfer RNA (tRNA)-based therapies to cure everything from Dravet syndrome and other neurological conditions to cardiology indications and muscular dystrophies.
Disappointing top-line data from two failed phase III studies of soticlestat for Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) have crumpled the stock of co-developer Ovid Therapeutics Inc., which lost three-quarters of its value June 17.
Disappointing top-line data from two failed phase III studies of soticlestat for Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) have crumpled the stock of co-developer Ovid Therapeutics Inc., which lost three-quarters of its value June 17.
