Neurology and epilepsy
Clinical research in Dravet syndrome with attention to major motor seizures, including hemiclonic seizures, focal seizures with motor signs, focal to bilateral tonic-clonic seizures, generalized tonic-clonic seizures, tonic seizures, tonic-atonic drop attacks, and bilateral clonic seizures.
- Seizure frequency
- Motor seizure control
- Dravet syndrome
Research activity in this area is centered on therapeutic options for severe childhood-onset epileptic encephalopathy.
Ophthalmology and optic nerve disorders
Studies in autosomal dominant optic atrophy focus on visual pathway disease and inherited optic nerve degeneration, with interest in treatment-related safety and systemic exposure.
- Autosomal dominant optic atrophy
- Optic nerve degeneration
- Visual impairment
The research profile includes conditions affecting central visual function and inherited neuro-ophthalmic disease.
Genetic medicine and rare disease therapeutics
The sponsor’s trial portfolio reflects work in genetic disorders with monogenic disease mechanisms, including neurologic and ophthalmic indications.
- Inherited disease
- Monogenic disorders
- Rare disease treatment
Current research spans conditions where targeted therapeutic development is directed at underlying genetic causation.
Clinical pharmacology and tolerability assessment
Ongoing studies include evaluation of safety, tolerability, and systemic exposure in both neurologic and ophthalmic patient populations.
- Safety monitoring
- Tolerability
- Exposure assessment
This area links therapeutic development with clinical characterization of treatment effects in central nervous system and eye disease settings.
