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The use of everolimus in the treatment of neurocognitive problems in tuberous sclerosis (TRON): study protocol for a randomised controlled trial.

Author: AngelLianna, Cannings-JohnRebecca, DaviesD Mark, DrewCheney, HoodKerry, KirbyNigel, McDermottEmer, McNamaraRachel, Owen-JonesEleri, RandellElizabeth, SampsonJulian R, SaxenaAnurag, SmalleyMichelle, StockwellLaura, de VriesPetrus J

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

Tuberous sclerosis complex (TSC) is a rare genetic disorder affecting 1 in 6,000 people, causing tumors in various organs and neurological problems. Most people with TSC experience TSC-associated neuropsychiatric disorders (TAND) that significantly impact their daily lives, education, work, and social interactions. Improved treatments for TAND could reduce the burden on patients, families, and the healthcare system.
Paper Details　
Original Abstract of the Article :
Tuberous sclerosis complex (TSC) is a genetic disorder affecting about 1 in 6000 people and is characterised by the development of tumours in many organs, including the skin and kidneys, and by a range of neurological and neuropsychiatric manifestations. TSC-associated neuropsychiatric disorders (TA...See full text at original site
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引用元：
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4981993/

データ提供：米国国立医学図書館（NLM）

A New Treatment Approach for Tuberous Sclerosis

Tuberous sclerosis complex (TSC) is a rare genetic disorder characterized by the growth of non-cancerous tumors in various organs, including the brain. These tumors can cause a wide range of neurological and neuropsychiatric issues, significantly impacting the lives of patients and their families. This research explores the potential of a new treatment approach using everolimus, a drug that inhibits the growth of these tumors.

Targeting Tumor Growth: Everolimus as a Potential Treatment

The TRON study is a randomized controlled trial designed to evaluate the efficacy and safety of everolimus in treating neurocognitive problems associated with TSC. The goal is to determine if everolimus can effectively reduce the size and impact of these tumors, leading to improvements in cognitive function and overall quality of life.

Hope for a Better Future: Potential Benefits for TSC Patients

If proven successful, everolimus could offer a new treatment option for individuals living with TSC, potentially alleviating the debilitating neuropsychiatric symptoms and improving their ability to participate in daily activities. Think of everolimus as a compassionate oasis in the challenging desert of TSC, providing hope for a brighter future for affected individuals.

Dr.Camel's Conclusion

This research represents a significant step forward in the treatment of TSC. By investigating the potential benefits of everolimus, we're gaining valuable insights into the underlying mechanisms of this complex disorder and exploring new therapeutic approaches. This research, like a beacon in the desert, shines a light on the possibilities for improved care and a better quality of life for individuals with TSC.

Date :
  1. Date Completed 2018-02-06
  2. Date Revised 2018-11-13
Further Info :

Pubmed ID

27515417

DOI: Digital Object Identifier

PMC4981993

SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

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