Tuberous Sclerosis
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EMA approval · 2026-08-06EMA Authorised: Hyftor (sirolimus) — Plusultra pharma GmbH
Sirolimus (Hyftor) from Plusultra pharma GmbH has been authorised for use in angiofibroma and tuberous sclerosis, designated as an orphan me…
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Role of Genetic Factors in the Development of Lung Disease
NHLBI-backed clinical trial is recruiting to study the role of genetic factors in the development of lung disease, including cystic fibrosis…
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EMA approval · 2025-11-20EMA Authorised: Votubia (everolimus) — Novartis Europharm Limited
The European Medicines Agency authorised Votubia (everolimus) for use in tuberous sclerosis, with Novartis Europharm Limited as the marketin…
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CLINICAL_TRIALS trial_result · 2025-05-18Clinical Trial Update: Reproductive Options in Inherited Skin Diseases
A clinical trial update indicates a study on reproductive options for inherited skin diseases is ongoing at Maastricht University Medical Ce…
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CLINICAL_TRIALS research · 2025-05-18Clinical Trial Update: Qualitative Study in Patients With Genodermatoses and Healthcare Professionals on Reproductive Counselling
Maastricht University Medical Center is conducting a qualitative study to explore reproductive counseling for patients with genodermatoses a…
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CLINICAL_TRIALS trial_result · 2023-08-14Clinical Trial Update: Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex [Phase 2, Phase 3]
Phase 2/3 dose-ranging study evaluating topical rapamycin cream for facial angiofibroma in tuberous sclerosis complex has completed with res…
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CLINICAL_TRIALS trial_result · 2023-04-04Clinical Trial Update: Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B) [Phase 2]
Phase 2 clinical trial on adjunctive ganaxolone for tuberous sclerosis complex (TSC) completed with long-term treatment data posted.
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CLINICAL_TRIALS trial_result · 2020-10-26Clinical Trial Update: Topical Rapamycin to Erase Angiofibromas in TSC [Phase 2]
Phase 2 trial evaluating topical rapamycin to reduce facial angiofibromas in tuberous sclerosis complex completed with results posted.
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CLINICAL_TRIALS trial_result · 2019-02-20Clinical Trial Update: Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex [Phase 3]
This entry reports the long-term results of a Phase 3 trial of topical sirolimus for angiofibromas in patients with tuberous sclerosis compl…
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CLINICAL_TRIALS withdrawal · 2018-04-30Clinical Trial Update: Turmeric as Treatment in Epilepsy [NA]
A clinical trial evaluating turmeric as a treatment for epilepsy has been withdrawn, including conditions such as Dravet syndrome, Lennox-Ga…
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CLINICAL_TRIALS trial_result · 2018-01-30Clinical Trial Update: A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous Sclerosis [Phase 2]
Phase 2 clinical trial evaluating everolimus for neurocognitive problems in tuberous sclerosis.
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CLINICAL_TRIALS trial_result · 2012-05-01Clinical Trial Update: Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1) [Phase 3]
Phase 3 EXIST-1 trial evaluating everolimus for subependymal giant cell astrocytoma in tuberous sclerosis complex demonstrates efficacy and …