” We have made
The EMBOLD study is a randomized, double-blind, placebo-controlled Phase 2 clinical trial to evaluate the safety, tolerability, efficacy (motor seizure frequency), and pharmacokinetics (PK) of
SCN2A-DEE is a monogenic seizure disorder caused by a variant of the SCN2A gene. The SCN2A gene is essential for the formation of sodium channel proteins in the brain, which control the flow of sodium ions into neurons. This movement of sodium ions is a major part of generating electrical signals called action potentials, the way cells communicate. SCN2A-DEE exhibits a wide range of phenotypes. Early-onset SCN2A-DEE presents before three months and can have a profound impact on patients, including drug-resistant seizures, significant cognitive impairment, movement disorders such as dystonia or ataxia, and in other body systems such as gastrointestinal or ocular. There is currently no approved treatment for SCN2A-DEE, and the standard of care usually involves a regimen of many concurrent antiepileptic drugs as well as drugs for comorbidities. Despite these interventions, more than 70% of early-onset SCN2A-DEE patients live with uncontrolled seizures and approximately 75% live with severe intellectual disability.
SCN8A-DEE is a rare developmental and epileptic encephalopathy caused by a variant of the SCN8A gene. The SCN8A gene is essential for the formation of sodium channel proteins in the brain, which control the tracking of sodium ions in neurons. This movement of sodium ions is a major part of generating electrical signals called action potentials, the way cells communicate. Patients suffer from recurrent, usually drug-resistant seizures that begin on the first day of life. Seizures can be of several different types, up to dozens per day, with poor response to current treatment options. Patients with SCN8A-DEE have significant cognitive impairment, ranging from moderate to severe; often movement disorders, such as dystonia or ataxia; and problems in other bodily systems such as gastrointestinal or ocular. SCN8A-DEE patients may also exhibit autonomic features such as increases or decreases in heart rate, abnormal breathing, and cyanosis.
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Alex Kane Praxis Precision Medicinesinvestors@praxismedicines.com 617-300-8481 Media Contact: Ian Stone Canale CommunicationsIan.email@example.com 619-849-5388
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