Achondroplasia
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CLINICAL_TRIALS trial_result · 2025-10-22Clinical Trial Update: Study of Infigratinib in Children With Achondroplasia [Phase 2]
This update notes that the Phase 2 trial of Infigratinib in children with achondroplasia has been completed.
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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 trial_result · 2025-02-25Clinical Trial Update: Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia [Phase 2]
Phase 2 study evaluating SAR442501 in pediatric achondroplasia assessing safety, tolerability, pharmacokinetics/pharmacodynamics, and effica…
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FDA approval · 2024-11-04FDA Approved: VOXZOGO 0.4MG — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved BioMarin’s supplemental application to add a 0.4 mg strength of VOXZOGO (vosoritide) for achondroplasia.
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CLINICAL_TRIALS trial_result · 2024-10-17Clinical Trial Update: Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
Pfizer-sponsored observational study on clinical and anthropometric features of children with Achondroplasia was terminated, with results po…
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CLINICAL_TRIALS trial_result · 2024-09-20Clinical Trial Update: A Study to Learn About Recifercept in Patients With Achondroplasia
Pfizer completed a clinical trial evaluating Recifercept in achondroplasia. Results from the study were posted on ClinicalTrials.gov in Sept…
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CLINICAL_TRIALS trial_result · 2024-06-13Clinical Trial Update: A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia [Phase 2]
Phase 2 trial results for BMN-111 (vosoritide) in infants and young children with achondroplasia have been completed and results posted.
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CLINICAL_TRIALS trial_result · 2024-03-07Clinical Trial Update: A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia [Phase 2]
A Phase 2 dose-escalation study of once-weekly TransCon CNP in prepubertal children with achondroplasia has completed, and results have been…
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CLINICAL_TRIALS trial_result · 2024-02-15Clinical Trial Update: A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia [Phase 2]
Phase 2 trial update evaluating safety, tolerability, and effectiveness of Recifercept in children with Achondroplasia; study terminated wit…
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CLINICAL_TRIALS trial_result · 2024-02-08Clinical Trial Update: Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia [Phase 2]
A Phase 2 continuation study of Recifercept in achondroplasia has terminated; results have been posted.
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CLINICAL_TRIALS trial_result · 2023-11-02Clinical Trial Update: A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia [Phase 2]
A Phase 2 dose-escalation trial is evaluating safety, efficacy, and pharmacokinetics of weekly subcutaneous TransCon CNP in children with Ac…
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FDA approval · 2023-10-20FDA Approved: VOXZOGO 0.4MG — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approves BioMarin's supplemental application for VOXZOGO 0.4 mg (vosoritide), adding a 0.4 mg dosage strength to the labeling for achond…
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CLINICAL_TRIALS trial_result · 2023-02-28Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia [Phase 4]
Phase 4 clinical trial evaluating recombinant human growth hormone in children with achondroplasia to assess efficacy and safety.
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CLINICAL_TRIALS trial_result · 2022-03-02Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia [Phase 3]
Phase 3 trial evaluating BMN 111 in children with Achondroplasia has completed with results posted.
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FDA approval · 2021-11-19FDA Approved: VOXZOGO 0.4MG — Original Application (BioMarin Pharmaceutical Inc.)
FDA approved VOXZOGO (vosoritide) 0.4 mg under an original application from BioMarin for the treatment of achondroplasia in pediatric patien…
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EMA approval · 2021-06-25New treatment for people with dwarfism
EMA announces a new treatment option for people with dwarfism, indicating regulatory approval or a positive recommendation for a therapy add…
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CLINICAL_TRIALS trial_result · 2021-01-15Clinical Trial Update: A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia [Phase 2]
Phase 2 trial evaluating BMN 111 in children with achondroplasia has completed with posted results.
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CLINICAL_TRIALS trial_result · 2017-05-05Clinical Trial Update: A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With Somatropin
A completed clinical trial update describing a survey to collect data on adult height in people with achondroplasia treated with somatropin.
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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 trial_result · 2015-04-14Clinical Trial Update: C-Type Natriuretic Peptide and Achondroplasia
This update reports the completion of a clinical trial investigating C-type natriuretic peptide for achondroplasia and hypochondroplasia.
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CLINICAL_TRIALS trial_result · 2012-06-11Clinical Trial Update: A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers [Phase 1]
Phase 1 trial evaluating safety and tolerability of BMN 111 (vosoritide) in healthy adult volunteers has completed; results summarize safety…
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CLINICAL_TRIALS trial_result · 2011-04-04Clinical Trial Update: Simultaneous Lengthening of Bilateral Femora and Tibiae in Achondroplastic Patients [NA]
A clinical trial investigating simultaneous lengthening of the bilateral femora and tibiae in achondroplasia patients has completed.
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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…