Dravet syndrome

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Phase 2 ELEKTRA Study of Soticlestat (TAK-935/OV935) Meets Primary Endpoint Reducing Seizure Frequency in Children with Dravet Syndrome or Lennox-Gastaut Syndrome Nasdaq:OVID Myoclonic Seizures, Absence Seizures, Dravet Syndrome, New York March, X Chromosome, Pediatric Physical Therapy, Genetic Mutation, Rare Disease, Neurological Disorders

Primary endpoint achieved, demonstrating a statistically significant reduction of seizures from baseline compared to placebo (p=0.0007) in the combined...

Sexually Divergent Mortality and Partial Phenotypic Rescue After Gene Therapy in a Mouse Model of Dravet Syndrome | Human Gene Therapy Divergent, Dravet Syndrome, Gene Therapy, Genetic Disorders, Genetic, A Mouse, Womens Health, Human

Dravet syndrome (DS) is a neurodevelopmental genetic disorder caused by mutations in the SCN1A gene encoding the α subunit of the NaV1.1 voltage-gated sodium channel that controls neuronal action potential firing. The high density of this mutated channel in GABAergic interneurons results in impaired inhibitory neurotransmission and subsequent excessive activation of excitatory neurons. The syndrome is associated with severe childhood epilepsy, autistic behaviors, and sudden unexpected death…

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