Achondroplasia
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Study of TYRA-300 in Children With Achondroplasia: BEACH301 [Phase 2]
A Phase 2 study of TYRA-300 in children with achondroplasia (BEACH301) is currently recruiting.
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CLINICAL_TRIALS research · 2026-04-22Clinical Trial Update: Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia [Phase 2, Phase 3]
A Phase 2/3 trial is evaluating BMN 333 versus Vosoritide to assess efficacy and safety in children with achondroplasia; the study is curren…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia [Phase 2]
A Phase 2 trial in infants (0 to <2 years) with achondroplasia evaluating the efficacy and safety of TransCon CNP vs placebo; recruiting sta…
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: Achondroplasia Natural History Multicenter Clinical Study
This is an ongoing multicenter natural history study of achondroplasia. Participant recruitment is currently underway.
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) [Phase 2]
Phase 2 interventional study evaluating infigratinib for achondroplasia in children under 3 years old; currently recruiting.
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia [Phase 2]
The trial to evaluate the safety of vosoritide in at-risk infants with achondroplasia remains active but not recruiting.
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) [Phase 2]
Phase 2 trial update for BMN 111 (vosoritide) evaluating long-term safety, tolerability, and efficacy in children with achondroplasia. The s…
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CLINICAL_TRIALS research · 2026-02-20Clinical Trial Update: Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
Prospective, monocentric study sponsored by SYSNAV to measure limb movement in patients with FGFR3-related skeletal dysplasia. Recruitment i…
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CLINICAL_TRIALS research · 2026-02-18Clinical Trial Update: A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia [Phase 2]
Phase 2 clinical trial evaluating navepegritide in combination with lonapegsomatropin for children with achondroplasia; current status is ac…
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CLINICAL_TRIALS research · 2025-12-24Clinical Trial Update: A Study in Children With Achondroplasia
Abbisko Therapeutics is recruiting for a pediatric study in children with achondroplasia.
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CLINICAL_TRIALS research · 2025-10-31Clinical Trial Update: Extension Study of Infigratinib in Children With Achondroplasia (ACH) [Phase 2]
An extension study of Infigratinib for children with achondroplasia is in Phase 2 and currently enrolling by invitation. The study is sponso…
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CLINICAL_TRIALS research · 2025-10-27Clinical Trial Update: A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia [Phase 2, Phase 3]
This entry notes an ongoing Phase 2/Phase 3 study of TransCon CNP for Achondroplasia in children and adolescents. It is assessing long-term …
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CLINICAL_TRIALS research · 2025-04-29Clinical Trial Update: Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study） [Phase 3]
Phase 3 AOBA study evaluating KK8398 in Achondroplasia is currently recruiting; the trial aims to assess efficacy and safety.
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CLINICAL_TRIALS research · 2017-02-27Clinical Trial Update: Special Survey for Long Term Application
The document announces the completion of a long-term special survey for Novo Nordisk's clinical trial NCT01516229 in patients with Achondrop…
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CLINICAL_TRIALS research · 2008-03-04Clinical Trial Update: Issues Surrounding Prenatal Genetic Testing for Achondroplasia
This clinical trial update explores issues surrounding prenatal genetic testing for achondroplasia. It was sponsored by the National Human G…