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A combination of ascorbic acid and α-tocopherol to test the effectiveness and safety in the fragile X syndrome: study protocol for a phase II, randomized, placebo-controlled trial.

Author: Caballero-AndaluzRafaela, Calvo-MedinaRocio, Fernández-CarvajalIsabel, Ferrando-LucasTeresa, García-GuiradoFrancisco, Pérez-CostillasLucia, Quintero-NavarroCarolina, Sánchez-SalidoLourdes, de Diego-OteroYolanda, del Arco-HerreraIgnacio

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

Fragile X syndrome (FXS) is a genetic condition that affects both boys and girls, causing developmental delays, behavioral problems, and physical and neurological issues. This study examines whether a combination of vitamin C and vitamin E is effective in reducing symptoms of FXS in boys ages 6-18, compared to a placebo. The study will also assess the safety of the treatment and look for changes in behavior, cognitive function, blood levels, and how the treatment affects home and school settings.
Paper Details　
Original Abstract of the Article :
Fragile X syndrome (FXS) is an inherited neurodevelopmental condition characterised by behavioural, learning disabilities, physical and neurological symptoms. In addition, an important degree of comorbidity with autism is also present. Considered a rare disorder affecting both genders, it first beco...See full text at original site
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引用元：
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4168067/

データ提供：米国国立医学図書館（NLM）

Fragile X Syndrome: A Desert Oasis of Potential Treatments

Fragile X syndrome (FXS), a neurodevelopmental disorder, is a challenging desert landscape of cognitive and behavioral difficulties. This study, akin to a research expedition seeking a path to alleviate suffering, investigates the potential of a combination of ascorbic acid (vitamin C) and α-tocopherol (vitamin E) to improve symptoms in individuals with FXS. It's a glimmer of hope in the search for effective treatments for this debilitating condition.

A New Oasis of Hope for Fragile X Syndrome

The study aims to evaluate the efficacy and safety of this combination therapy, hoping to improve the lives of individuals with FXS. It's a testament to the ongoing quest for new treatments, a journey fueled by compassion and a desire to ease the burdens of those affected by neurodevelopmental disorders.

Navigating the Complexities of Neurodevelopmental Disorders

This research highlights the importance of exploring novel therapeutic approaches for complex neurodevelopmental disorders. By understanding the underlying mechanisms of FXS, researchers can develop targeted interventions that address the specific needs of individuals affected by this condition, offering a path to better outcomes.

Dr. Camel's Conclusion

This study represents a significant step forward in the search for effective treatments for Fragile X syndrome. It's a reminder that even in the vast desert of medical unknowns, there is always hope for new discoveries that can alleviate suffering and improve the lives of those affected by complex disorders.
Date :
  1. Date Completed 2015-05-28
  2. Date Revised 2021-10-21
Further Info :

Pubmed ID

25187257

DOI: Digital Object Identifier

PMC4168067

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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