Developmental and epileptic encephalopathies, the most severe group of epilepsies, are
characterized by seizures and frequent epileptiform activity associated with developmental …

The era of RNA medicine has become a reality with the success of messenger RNA (mRNA)
vaccines against COVID-19 and the approval of several RNA interference (RNAi) agents in …

Variants in KCNT1, encoding a sodium-gated potassium channel (subfamily T member 1),
have been associated with a spectrum of epilepsies and neurodevelopmental disorders …

A number of mutations to members of several CNS potassium (K) channel families have
been identified which result in rare forms of neonatal onset epilepsy, or syndromes of which …

In recent years, there has been a significant increase in preclinical studies to test genetic
therapies for epilepsy. Some of these therapies have advanced to clinical trials and are …

The process of develo** therapies to treat rare diseases is fraught with financial,
regulatory, and logistical challenges that have limited our ability to build effective treatments …

Ogden syndrome, also known as NAA10‐related neurodevelopmental syndrome, is a rare
genetic condition associated with pathogenic variants in the NAA10 N‐terminal acetylation …

Intracerebroventricular (ICV) administration is increasingly being explored as a means for
delivering antiseizure and antiepileptic therapies to epileptic brain tissue. This route …

Objective Pathogenic variants in KCNT2 are rare causes of developmental epileptic
encephalopathy (DEE). We herein describe the phenotypic and genetic features of patients …

Objective KCNT1‐related epilepsies encompass three main phenotypes:(i) epilepsy of
infancy with migrating focal seizures (EIMFS),(ii) autosomal dominant or sporadic sleep …