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New drug aims to help kids with rare genetic short stature grow taller

NCT ID NCT04219007

First seen Apr 28, 2026 · Last updated Jun 17, 2026 · Updated 10 times

Summary

This study tests a drug called vosoritide in 56 children with short stature caused by certain genetic conditions. The drug targets the growth plate to help children grow faster. Participants are observed for 6 months, then treated with daily injections for 12 months to check safety and growth improvement.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010, United States

Conditions

The condition(s) this trial relates to.

cardiofaciocutaneous syndrome Costello syndrome Dwarfism hypochondroplasia neurofibromatosis type 1 Noonan syndrome SHOX-related short stature spondyloepimetaphyseal dysplasia, aggrecan type

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.