ACHONDROPLASIA
Clinical trials for ACHONDROPLASIA explained in plain language.
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Could a daily injection spare babies with dwarfism from risky spine surgery?
Disease control OngoingThis study tests a drug called vosoritide in infants with achondroplasia (a common form of dwarfism) who are at high risk of needing spinal surgery. The goal is to see if the drug is safe and can help the skull opening grow larger, potentially reducing the need for surgery. About…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 26, 2026 05:57 UTC
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New hope for kids with dwarfism: daily pill may boost growth
Disease control ENROLLING_BY_INVITATIONThis study tests a daily pill called infigratinib in children with achondroplasia, the most common form of dwarfism. The goal is to see if it safely improves growth over several years. About 300 children who were in earlier studies or are new to the drug will take part. Because t…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2 • Sponsor: QED Therapeutics, a BridgeBio company • Aim: Disease control
Last updated May 26, 2026 05:55 UTC
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New drug shows promise for helping children with dwarfism grow
Disease control OngoingThis study tests a drug called BMN 111 in children with achondroplasia, a common form of dwarfism. The goal is to see if it safely helps them grow taller over several years. Participants must have completed a previous related study. The treatment aims to improve growth and daily …
Matched conditions: ACHONDROPLASIA
Phase: PHASE3 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 26, 2026 05:53 UTC
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Daily shot shows promise for helping kids with dwarfism grow taller
Disease control OngoingThis study tests a daily injection called BMN 111 in about 70 children with achondroplasia, the most common form of dwarfism. The goal is to see if the drug is safe and helps kids grow taller over several years until they reach near-adult height. All children in the study had alr…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 26, 2026 05:53 UTC
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New combo therapy aims to boost growth in kids with dwarfism
Disease control OngoingThis study tests whether two weekly injections, navepegritide and lonapegsomatropin, can safely improve growth in children aged 2 to 11 with achondroplasia (a common form of dwarfism). The trial involves 22 children and lasts 156 weeks. The goal is to see if the combination works…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2 • Sponsor: Ascendis Pharma Growth Disorders A/S • Aim: Disease control
Last updated May 22, 2026 14:01 UTC
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New drug shows promise for children with dwarfism in Long-Term trial
Disease control OngoingThis study looks at the long-term safety and effectiveness of a daily injection called BMN 111 in 30 children with achondroplasia, the most common form of dwarfism. Participants had already received two years of treatment in an earlier study. Researchers are monitoring side effec…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 22, 2026 13:53 UTC
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Weekly shot shows promise for helping kids with dwarfism grow
Disease control ENROLLING_BY_INVITATIONThis study tests a once-weekly injection called TransCon CNP in children and teens with achondroplasia, the most common form of dwarfism. The goal is to see if it is safe and helps improve growth over several years. Participants who were in a previous TransCon CNP trial can join,…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2, PHASE3 • Sponsor: Ascendis Pharma Growth Disorders A/S • Aim: Disease control
Last updated May 06, 2026 16:02 UTC