Turner Syndrome
Turner Syndrome is a genetic condition that affects females, where one of the X chromosomes is missing or partially missing, leading to short stature, delayed puberty, and various health issues.
Vosoritide for Growth Issues in Children
We are studying a new treatment called vosoritide for children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome who haven't responded well to standard growth hormone therapy. The goal is to see if it helps improve their growth and is safe to use.
What diseases are being studied?
Health conditions and diseases that the clinical trial is designed to study and treat.
Short Stature Homeobox Gene Mutation
Short Stature Homeobox Gene Mutation refers to genetic changes that can lead to reduced height and growth issues, often requiring medical intervention for management.
Noonan Syndrome
Noonan Syndrome is a genetic disorder that can cause a variety of symptoms including distinctive facial features, short stature, and heart defects, affecting both males and females.
Who can participate in the clinical trial?
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
- Are you between 3 and 10 years old if you are female, or between 3 and 11 years old if you are male?
- Do you have a diagnosis of Turner syndrome, SHOX deficiency, or Noonan syndrome?
- Have you been receiving growth hormone treatment for at least 1 year?
- Are you currently in Tanner Stage 1 (the first stage of puberty)?
- Do you have any other serious health conditions that could cause short stature, like kidney or heart disease?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
C-Type Natriuretic Peptide
What similar medicines might you know?
Don't worry if you don't know the medicines tested in the trial. There is a chance you have heard about the similar medicines. Check the list below to see if you are familiar with any of them.
Genotonorm
Genotonorm
Genotonorm
Where is the clinical trial being conducted?
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
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Centre Hospitalier Universitaire D'Angers
Pediatric Endocrinology & Diabetology
Angers, France
Hopital Des Enfants
Endocrinology, Gynecology & Medical Genetics
Blagnac, France
Bicetre Hospital
Pediatric Endocrinology & Diabetes for Children
Croulebarbe, France
Sponsor: Biomarin Pharmaceutical Inc.
Last updated: Nov 26, 2025
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.