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C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia.

Author: BacinoCarlos A, BostwickBret, ChanMing-Liang, CharrowJoel, CherukuriAnu, Cormier-DaireValerie, DayJonathan, DicksonPatricia, HarmatzPaul, Hoover-FongJulie, Huntsman LabedAlice, IrvingMelita, JayaramKala, JehaGeorge S, LarimoreKevin, Le Quan SangKim-Hanh, OwenNatalie, PhillipsJohn, SavarirayanRavi

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

A phase 2 dose-finding study and extension study in children with achondroplasia found that vosoritide, a biologic analogue of C-type natriuretic peptide, was associated with a generally mild side-effect profile and resulted in a sustained increase in annualized growth velocity for up to 42 months.
Paper Details　
Original Abstract of the Article :
BACKGROUND: Achondroplasia is a genetic disorder that inhibits endochondral ossification, resulting in disproportionate short stature and clinically significant medical complications. Vosoritide is a biologic analogue of C-type natriuretic peptide, a potent stimulator of endochondral ossification. ...See full text at original site
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引用元：
https://doi.org/10.1056/NEJMoa1813446

データ提供：米国国立医学図書館（NLM）

Vosoritide: A New Oasis in the Desert of Achondroplasia

Achondroplasia is a genetic disorder that affects bone growth, resulting in disproportionate short stature. It's like navigating a challenging landscape, where the paths to normal growth are blocked by genetic roadblocks. This research investigates the potential of vosoritide, a biologic analogue of C-type natriuretic peptide, as a treatment option for children with achondroplasia. The researchers conducted a multinational, phase 2, dose-finding study and extension study, evaluating the safety and efficacy of vosoritide in children with achondroplasia. They found that vosoritide was generally well tolerated and resulted in a sustained increase in growth velocity in children with achondroplasia.

Vosoritide: A Beacon of Hope for Achondroplasia

This research provides a ray of hope for children with achondroplasia, offering a potential pathway to improved growth and development. The study's findings suggest that vosoritide could be a valuable tool for managing achondroplasia. It's like finding a hidden spring in the desert of growth disorders, offering a new source of nourishment and potential for growth.

Navigating the Desert of Growth Disorders: A Journey Towards a Brighter Future

This research emphasizes the importance of ongoing research and development of innovative treatments for achondroplasia. It encourages continued exploration of new therapies that can improve the lives of children with this challenging condition. This journey through the desert of growth disorders requires a commitment to finding new oases of knowledge and treatment strategies, offering a brighter future for those affected by this condition.

Dr.Camel's Conclusion

This study provides a promising glimpse into the potential of vosoritide for treating achondroplasia. It's a reminder that even in the seemingly barren desert of rare diseases, there are opportunities to find new oases of hope and treatment. This research is a step towards a more effective and personalized approach to managing achondroplasia, offering a brighter future for children with this challenging condition.

Date :
  1. Date Completed 2019-07-29
  2. Date Revised 2019-09-10
Further Info :

Pubmed ID

31269546

DOI: Digital Object Identifier

10.1056/NEJMoa1813446

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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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