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Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study.

Author: AlanayYasemin, ArundelPaul, BacinoCarlos A, BaselDonald, BoberMichael B, CharrowJoel, DayJonathan R S, FishelevaElena, FontRosendo Ullot, GinebredaIgnacio, HarmatzPaul, HoernschemeyerDaniel, Hoover-FongJulie, Huntsman-LabedAlice, IrvingMelita, JayaramKala, KotaniYumiko, Leiva-GeaAntonio, Luna-GonzálezFelipe, MochizukiHiroshi, MohnikeKlaus, OzonoKeiichi, PolgreenLynda E, PorcoDania M, RutschFrank, SaalHoward M, SavarirayanRavi, ToftsLouise, WhiteKlane K, WilcoxWilliam R, YasuiNatsuo

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
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

This phase 3 extension study documented the efficacy and safety of continuous, daily vosoritide treatment in children with achondroplasia for two years.
Paper Details　
Original Abstract of the Article :
Achondroplasia is caused by pathogenic variants in the fibroblast growth factor receptor 3 gene that lead to impaired endochondral ossification. Vosoritide, an analog of C-type natriuretic peptide, stimulates endochondral bone growth and is in development for the treatment of achondroplasia. This ph...See full text at original site
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引用元：
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8327889/

データ提供：米国国立医学図書館（NLM）

Vosoritide: A Potential Treatment for Achondroplasia

Achondroplasia is a rare genetic disorder that affects bone growth. This study investigates the long-term effects of vosoritide, a C-type natriuretic peptide analog, in children with achondroplasia. The researchers conducted a two-year, open-label extension study to evaluate the efficacy and safety of vosoritide for the treatment of this condition.

Vosoritide: A Potential Solution for Achondroplasia

The study found that vosoritide had persistent growth-promoting effects in children with achondroplasia, suggesting that it could be a valuable treatment option for this debilitating condition. It's like finding a spring of hope in the desert of rare diseases – vosoritide might be a valuable source of relief for those affected by achondroplasia.

Long-Term Benefits: Continued Research Needed

The study's findings are promising, but it emphasizes the need for further research to fully understand the long-term effects of vosoritide. Continued monitoring and evaluation are crucial to ensure the safety and efficacy of this potential treatment. As we journey through the desert of rare diseases, we need to carefully assess each potential solution, ensuring that they are safe and effective for those who need them.

Dr. Camel's Conclusion

This study explores the use of vosoritide as a potential treatment for achondroplasia. The findings suggest that vosoritide could offer a valuable treatment option for this rare disease. It's an exciting discovery that could bring hope to families affected by achondroplasia. Further research is essential to ensure the safety and efficacy of this potential therapy, but the results are promising.

Date :
  1. Date Completed 2022-03-22
  2. Date Revised 2022-05-31
Further Info :

Pubmed ID

34341520

DOI: Digital Object Identifier

PMC8327889

Related Literature

Article Analysis
・Effects of vosoritide
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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