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Alpelisib for treatment of patients with PIK3CA-related overgrowth spectrum (PROS).

Author: AdamsDenise M, AnkrahNii, BranleFabrice, CanaudGuillaume, HansfordJordan R, IrvineAlan D, Lopez GutierrezJuan Carlos, O'ConnellPaul, PapadimitriouAthanasia, RidolfiAntonia, TurnerStuart, VabresPierre

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Overview

Alpelisib, a PI3Kα-selective inhibitor, is a novel therapeutic approach for treating PIK3CA-related overgrowth spectrum (PROS). While further studies are ongoing, preliminary data suggest its safety and efficacy in pediatric and adult patients with PROS.
Paper Details　
Original Abstract of the Article :
PIK3CA-related overgrowth spectrum (PROS) encompasses several rare conditions resulting from activating variants in PIK3CA. Alpelisib, a PI3Kα-selective inhibitor, targets the underlying etiology of PROS, offering a novel therapeutic approach to current management strategies. This study evaluated th...See full text at original site
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引用元：
https://doi.org/10.1016/j.gim.2023.100969

データ提供：米国国立医学図書館（NLM）

Alpelisib: A New Dawn for Treating PIK3CA-Related Overgrowth Spectrum

Medical research is a continuous journey of exploration, seeking new solutions for the complexities of human health. This study delves into the treatment of PIK3CA-related overgrowth spectrum (PROS), a group of rare conditions characterized by excessive growth. The researchers evaluated the safety and efficacy of alpelisib, a PI3Kα-selective inhibitor, in patients with PROS. They aimed to understand how this drug, specifically targeting the underlying cause of PROS, could impact the management of these conditions. The results of the study suggest that alpelisib holds promise as a novel treatment option for PROS, offering a potential alternative to existing management strategies.

A New Approach to Managing Overgrowth Conditions

The research presents a promising new direction for treating PROS. Alpelisib's targeted approach, addressing the root cause of the condition, could significantly impact the lives of patients with PROS. Further research is needed to fully understand its long-term effects and optimize its use in clinical practice.

Navigating the Uncharted Territories of Rare Diseases

This study highlights the importance of research into rare diseases. Finding effective treatments for these conditions can significantly improve the lives of those affected. The development of alpelisib offers hope for patients with PROS and underscores the ongoing quest for new therapies in the field of rare diseases.

Dr.Camel's Conclusion

Alpelisib presents a novel and potentially effective treatment option for PIK3CA-related overgrowth spectrum. This research highlights the crucial role of targeted therapies in managing rare diseases and underscores the ongoing efforts to provide better care for those affected.
Date :
  1. Date Completed n.d.
  2. Date Revised 2023-12-09
Further Info :

Pubmed ID

37634128

DOI: Digital Object Identifier

10.1016/j.gim.2023.100969

Related Literature

Article Analysis
・Effects of alpelisib
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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