ACHONDROPLASIA
Clinical trials for ACHONDROPLASIA explained in plain language.
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Breakthrough drug boosts growth in kids with dwarfism
Disease control OngoingThis study tests a medicine called BMN 111 in children with achondroplasia, the most common form of dwarfism. The goal is to see if it safely helps them grow taller and improves their daily life. Children who completed a previous study can join, and they will receive the drug for…
Matched conditions: ACHONDROPLASIA
Phase: PHASE3 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 17, 2026 08:04 UTC
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New drug combo aims to boost growth in kids with dwarfism
Disease control OngoingThis study tests whether giving two weekly injections together can improve growth in children with achondroplasia, a common form of dwarfism. About 22 children aged 2 to 11 will receive the combination for up to 156 weeks. The goal is to see if the drugs work better together than…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2 • Sponsor: Ascendis Pharma Growth Disorders A/S • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
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New drug shows promise for helping children with dwarfism grow
Disease control OngoingThis study looks at the long-term safety and effectiveness of a daily injection called BMN 111 in children with achondroplasia, a common form of dwarfism. About 30 children who already received BMN 111 for two years in a previous study will continue treatment. Researchers will tr…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 15, 2026 11:54 UTC
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New hope for children with dwarfism: Long-Term drug trial launches
Disease control ENROLLING_BY_INVITATIONThis study tests the long-term safety and effectiveness of an oral drug called infigratinib in children with achondroplasia, the most common form of dwarfism. About 300 children who previously took part in a related study or are new to the treatment will receive the drug for seve…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2 • Sponsor: QED Therapeutics, a BridgeBio company • Aim: Disease control
Last updated May 15, 2026 11:53 UTC
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Daily shot shows promise for helping kids with dwarfism grow
Disease control OngoingThis study looks at the long-term safety and effectiveness of a daily injection called BMN 111 in children with achondroplasia, the most common form of dwarfism. About 73 children who completed a previous year-long study will receive the drug until they reach near-adult height. T…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 12, 2026 13:41 UTC
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Could this drug help babies with dwarfism avoid risky brain 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 brain surgery due to a narrow skull opening. The main goal is to see if the drug is safe and if it can help the skull opening grow larger, potentia…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 11, 2026 20:49 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