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Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis.

Author: GroganDonna R, JudgeDaniel P, MerliniGiampaolo, ObiciLaura, PackmanJeff, PerliniStefano, Planté-BordeneuveViolaine, SchmidtHartmut, TrippTara

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.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

Tafamidis, a drug for the treatment of transthyretin (TTR) amyloidosis, effectively stabilized TTR associated with several non-Val30Met variants in a phase II study. Tafamidis 20 mg QD was well tolerated.
Paper Details　
Original Abstract of the Article :
This phase II, open-label, single-treatment arm study evaluated the pharmacodynamics, efficacy, and safety of tafamidis in patients with non-Val30Met transthyretin (TTR) amyloidosis. Twenty-one patients with eight different non-Val30Met mutations received 20 mg QD of tafamidis meglumine for 12 month...See full text at original site
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引用元：
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3838581/

データ提供：米国国立医学図書館（NLM）

Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis

This phase II study investigates the effects of tafamidis, a drug used to treat transthyretin amyloidosis, a rare and debilitating disease. Imagine transthyretin amyloidosis as a harsh desert landscape, where the buildup of amyloid protein causes damage to organs and tissues. The authors evaluated the effects of tafamidis on patients with non-Val30Met transthyretin amyloidosis, finding that it effectively stabilized the protein responsible for the disease. This research offers a beacon of hope for patients battling this rare and complex condition, potentially providing a new pathway for treatment.

Tafamidis: A Potential Oasis in the Desert of Transthyretin Amyloidosis

This phase II study provides encouraging evidence for the effectiveness of tafamidis in treating non-Val30Met transthyretin amyloidosis. This is a significant breakthrough, offering a potential treatment option for patients with this rare and debilitating disease.

Stabilizing the Protein: A Pathway to Treatment

This research highlights the importance of stabilizing the transthyretin protein in treating transthyretin amyloidosis. It's like finding a way to prevent the sands of the desert from shifting and causing damage. This study provides valuable insights into the potential of tafamidis as a treatment option for this rare and complex condition.

Dr.Camel's Conclusion

This phase II study provides encouraging evidence for the effectiveness of tafamidis in treating non-Val30Met transthyretin amyloidosis. It's like discovering a new oasis in the desert of this rare and debilitating disease, offering hope for patients and paving the way for further research and development of treatments.

Date :
  1. Date Completed 2014-07-21
  2. Date Revised 2021-10-21
Further Info :

Pubmed ID

24101373

DOI: Digital Object Identifier

PMC3838581

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Article Analysis
・Effects of tafamidis
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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