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The study will be comprised of 2 parts: Part A will evaluate the safety and pharmacokinetics of cannabidiol oral solution (OS) compared with SOC treatment for a period of 17 weeks in participants with tuberous sclerosis complex (TSC). The duration of Part A will be approximately 29 weeks total,
Rationale: Refractory epilepsy, meaning epilepsy that no longer responds to medication, is a common neurosurgical indication in children. Focal cortical dysplasia (FCD) is the most common cause. Tuberous sclerosis complex (TSC) is a rare genetic disease causing epilepsy in over 90% of affected
Infantile Spasms, historically call West Syndrome, is an infantile epileptic encephalopathy, typically occurring within the first two years of life. Infantile Spasms is often characterized by hypsarrhythmia on encephalogram (EEG) and developmental plateau or regression. It is estimated that
Background: Cannabidiol (CBD) and Δ9-tetrahydrocannabinol (THC) are the two major compounds found in the cannabis plant. Reports from patients, families, and the scientific community suggest that CBD (when used as an add-on therapy) decreases the number of convulsive seizures in children and adults
Seizures that are refractory to both medical and surgical therapy increase the risk of morbidity and mortality in children with epilepsy. At this point in time, options for these children are sparse and suboptimal. This hypothesis-driven phase 1 study aims to evaluate the use of a mammalian target
The clinical part of EPIMARKER is composed of 2 prospective studies (WP1 and WP2) of epilepsy progress in Tuberous Sclerosis Complex -TSC children, performed in 2 sites: Medical University of Warsaw -WUM and Children's Memoroal Health Institute - IPCZD, Poland.
WP1 and WP2 are designed to conduct
There is no optional treatment for patients with tuberous sclerosis complex (TSC) and refractory epilepsy.The investigator observed efficacy of aspirin in the treatment of in one child who got Kawasaki disease. Subsequent adjunctive aspirin therapy in four patients yielded a reducted frequency of
Questionnaire-based studies have shown that sleep problems occur in up to half of the children and a third of adults with tuberous sclerosis complex (TSC). However, there is only limited information on the nature of sleep problems and their impact on patients with TSC and their families. It is known
Tuberous sclerosis complex (TSC) is an autosomal dominant neurogenetic disorder, caused by heterozygous mutations in at least two different genes, TSC1, and TSC2. It is estimated to affect 1 in 6000, and demonstrates both phenotypic and genetic heterogeneity. It is characterized by a variety of
The central hypothesis of this Phase IIb trial is that early identification of electroencephalography (EEG) biomarkers and early treatment versus delayed treatment with vigabatrin in infants with tuberous sclerosis complex (TSC) will have a positive impact on developmental outcomes at 24 months of
Tuberous sclerosis complex (TSC) is an autosomal dominant disorder caused by mutations in tuberous sclerosis complex 1 (TSC1) or TSC2 tumor suppressor genes. TSC is characterized by tumors in a wide range of tissues, seizures, mental retardation, autism, and organ failure. Lymphangioleiomyomatosis
Tuberous Sclerosis Complex (TSC) is an autosomal dominant genetic disorder characterized by the growth of numerous tumors in different body parts related to dysregulation of the mechanistic target of rapamycin (mTOR) pathway. The overall incidence of TSC is estimated to be as high as 1 in 6000 to