Tuberous Sclerosis Complex
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2025-09-22Clinical Trial Update: TANDem-2: Closing the Gap to Interventions for TAND [NA]
Clinical trial update for TANDem-2, a study addressing interventions for TSC-associated neuropsychiatric disorders (TAND), is currently enro…
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CLINICAL_TRIALS research · 2025-08-29Clinical Trial Update: Gait in Rare Diseases
This clinical trial update indicates a study examining gait in rare diseases involving tuberous sclerosis complex and STXBP1 encephalopathy …
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FDA approval · 2025-08-06FDA Approved: ZTALMY — Supplemental Application (Immedica Pharma US Inc)
FDA approved a supplemental application for ZTALMY (ganaxolone) filed by Immedica Pharma US Inc.
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CLINICAL_TRIALS research · 2025-07-16Clinical Trial Update: Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders [Phase 2]
A Phase 2 trial is evaluating sertraline versus placebo for anxiety in children and adolescents with neurodevelopmental disorders, including…
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CLINICAL_TRIALS trial_result · 2025-07-11Clinical Trial Update: Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy [Phase 3]
A Phase 3 study evaluating adjunctive ganaxolone (GNX) versus placebo for tuberous sclerosis complex-related epilepsy has completed and resu…
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CLINICAL_TRIALS research · 2025-07-08Clinical Trial Update: TSC Biosample Repository and Natural History Database
Recruiting study to create a biosample repository and natural history database for tuberous sclerosis complex and lymphangioleiomyomatosis.
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EMA approval · 2025-07-04EMA Authorised: Hyftor (sirolimus) — Plusultra pharma GmbH
The EMA authorised Hyftor (sirolimus) by Plusultra pharma GmbH for angiofibroma and tuberous sclerosis complex with orphan medicine status.
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CLINICAL_TRIALS trial_result · 2025-06-04Clinical Trial Update: Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC [Phase 2]
A Phase 2 study of Basimglurant (NOE-101) in children, adolescents, and young adults with tuberous sclerosis complex has completed.
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CLINICAL_TRIALS trial_result · 2025-01-23Clinical Trial Update: Predicting Epileptogenic Tubers in Patients with Tuberous Sclerosis Complex
A completed clinical trial from Beijing Children's Hospital investigated predicting epileptogenic tubers in patients with tuberous sclerosis…
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CLINICAL_TRIALS trial_result · 2025-01-17Clinical Trial Update: The Effectiveness and Safety of Resective Epilepsy Surgery for TRE
This clinical trial update reports on the effectiveness and safety of resective epilepsy surgery for tuberous sclerosis complex–related epil…
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CLINICAL_TRIALS research · 2025-01-15Clinical Trial Update: Clinical Trial Data Set Re-use With Statistical Methodologies Tailored for Clinical Trials in Rare Diseases
This update describes a study on re-using clinical trial data with tailored statistical methods for rare diseases. The focus includes tubero…
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CLINICAL_TRIALS research · 2025-01-08Clinical Trial Update: Observational, Retrospective, Multicenter, Nonprofit Study on the Prevalence of Renal Involvement in Pediatric Patients With Tuberous Sclerosis
An observational, retrospective multicenter study to determine the prevalence of renal involvement in pediatric patients with tuberous scler…
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CLINICAL_TRIALS trial_result · 2024-12-19Clinical Trial Update: The Effectiveness and Safety of Vagus Nerve Stimulation for TRE
A completed clinical trial evaluated the effectiveness and safety of vagus nerve stimulation for tuberous sclerosis complex–associated epile…
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CLINICAL_TRIALS trial_result · 2024-11-22Clinical Trial Update: Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC [Phase 3]
Phase 3 clinical trial evaluating NPC-12Y Gel for skin lesions in Tuberous Sclerosis Complex has completed.
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CLINICAL_TRIALS trial_result · 2024-11-06Clinical Trial Update: KidneYou - Innovative Digital Therapy [NA]
Completed clinical trial update for KidneYou, an innovative digital therapy from Advice Pharma Group srl, investigating use in chronic kidne…
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CLINICAL_TRIALS trial_result · 2024-10-30Clinical Trial Update: TSC Remote Assessment and Intervention [NA]
Clinical trial update for Tuberous sclerosis complex remote assessment and intervention (NCT04698538) indicates the study is completed.
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CLINICAL_TRIALS research · 2024-10-26Clinical Trial Update: Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)
This clinical study investigates determinants of autism spectrum disorder and intellectual disability in tuberous sclerosis complex, and is …
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FDA approval · 2024-09-12FDA Approved: VIGADRONE — Supplemental Application (Upsher-Smith Laboratories, LLC)
The FDA approved a supplemental application for VIGADRONE (vigabatrin) by Upsher-Smith Laboratories, LLC under ANDA 210196.
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CLINICAL_TRIALS trial_result · 2024-08-19Clinical Trial Update: Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex [Phase 2]
A Phase 2 trial evaluated whether vigabatrin can prevent epilepsy in infants with tuberous sclerosis complex; the study results were posted …
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CLINICAL_TRIALS research · 2024-06-03Clinical Trial Update: Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1) [Phase 2]
This update describes the Phase 2 PRECISION 1 basket trial of nab-sirolimus for malignant solid tumors with pathogenic TSC1/TSC2 alterations…
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CLINICAL_TRIALS trial_result · 2024-05-16Clinical Trial Update: Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants [Phase 1, Phase 2]
This entry reports the completion of the Phase 1/2 trial Stopping TSC Onset and Progression 2, which studied epilepsy prevention in infants …
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FDA approval · 2024-03-04FDA Approved: EPIDIOLEX — Supplemental Application (Jazz Pharmaceuticals, Inc.)
The FDA approved a supplemental application for Epidiolex (cannabidiol) from Jazz Pharmaceuticals.
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CLINICAL_TRIALS trial_result · 2024-02-08Clinical Trial Update: Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants [Phase 2, Phase 3]
A clinical trial comparing rapamycin to vigabatrin for prevention of Tuberous Sclerosis Complex symptoms in infants, spanning Phase 2 and Ph…
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FDA approval · 2023-10-20FDA Approved: EPIDIOLEX — Supplemental Application (Jazz Pharmaceuticals, Inc.)
FDA approved a supplemental application for Epidiolex (cannabidiol) submitted by Jazz Pharmaceuticals, Inc. The action updates the labeling …
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CLINICAL_TRIALS trial_result · 2023-08-08Clinical Trial Update: Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex [Phase 3]
A Phase 3 placebo-controlled trial evaluating rapamycin for drug-resistant epilepsy in tuberous sclerosis complex is recruiting.