Tuberous Sclerosis Complex (TSC)
Our mission: passionately work to transform the lives of individuals with tuberous sclerosis complex.
At Marinus, we are committed to changing patients’ lives through innovative medicine and compassionate advocacy. We work hard to help ensure TSC patients’ stories are heard and their medical needs are understood.
What is Tuberous Sclerosis Complex?
Tuberous sclerosis complex (TSC) is a rare genetic disorder caused by inherited mutations in the TSC1 gene or TSC2 gene that can affect any or all systems of the body, resulting in non-malignant tumors in the brain, skin, kidney, heart, eyes, and lungs. The condition is a leading cause of genetic epilepsy, often occurring in the first year of life as either focal seizures or infantile spasms. While the disease phenotype can be extremely variable, neurologic manifestations such as epilepsy can be seen in up to 90% of TSC patients.
Tuberous sclerosis complex treatment options
There are currently limited disease-specific treatments approved for seizure types that occur in TSC patients. Despite these available treatments, many individuals affected by TSC continue to experience uncontrolled seizures and are in need of alternate therapeutic options.
TrustTSC Phase 3 Study**
** This study is no longer enrolling patients.
The TrustTSC (NCT05323734) trial is a global Phase 3 randomized, double-blind, placebo-controlled study of ganaxolone treatment in children and adults with tuberous sclerosis complex (TSC). The study is exploring whether treatment with investigational ganaxolone can reduce the frequency of seizures associated with TSC in children and adults.
TSC occurs with a frequency of 1 in 6,000, with a mutation found in 85% of patients.
More information about Marinus’ clinical trials can be found on ClinicalTrials.gov.
Learn more about tuberous sclerosis complex
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