Vosoritide Evidence: Trial Results and Peer-Reviewed Publications

Hipa.ai Research · Source: PubMed & ClinicalTrials.gov / AACT · Last updated:

The clinical evidence base for Vosoritide comprises 2 peer-reviewed publications across 2 journals, 0 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Dwarfism. Most recent publication: Vosoritide treatment for children with hypochondroplasia: a phase 2 trial., EClinicalMedicine, 2024.

Top peer-reviewed publications

Curated set of pivotal-trial result papers and recent publications in high-tier journals.

  1. Rationale, design, and methods of a randomized, controlled, open-label clinical trial with open-label extension to investigate the safety of vosoritide in infants, and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery.
    Savarirayan R, Irving M, Maixner W, et al. · Sci Prog · 2022
    PubMed: PMID 33761804 · NCT04554940 · Dwarfism
  2. Vosoritide treatment for children with hypochondroplasia: a phase 2 trial.
    Dauber A, Zhang A, Kanakatti Shankar R, et al. · EClinicalMedicine · 2024
    PubMed: PMID 38813446 · NCT04219007 · Dwarfism

Publications by year

2022–2024: 2 publications.

2022
1
2024
1

Publications by indication

Dwarfism (2)

  • Vosoritide treatment for children with hypochondroplasia: a phase 2 trial.
    EClinicalMedicine · 2024 · PMID 38813446 · NCT04219007
  • Rationale, design, and methods of a randomized, controlled, open-label clinical trial with open-label extension to investigate the safety of vosoritide in infants, and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery.
    Sci Prog · 2022 · PMID 33761804 · NCT04554940

Publications by journal

  • Sci Prog1 paper
  • EClinicalMedicine1 paper

All Vosoritide publications (2)

2024 (1 paper)

  1. Vosoritide treatment for children with hypochondroplasia: a phase 2 trial.
    Dauber A, Zhang A, Kanakatti Shankar R, et al. · EClinicalMedicine · 2024 · Derived
    PubMed: PMID 38813446 · NCT04219007 · Dwarfism

2022 (1 paper)

  1. Rationale, design, and methods of a randomized, controlled, open-label clinical trial with open-label extension to investigate the safety of vosoritide in infants, and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery.
    Savarirayan R, Irving M, Maixner W, et al. · Sci Prog · 2022 · Derived
    PubMed: PMID 33761804 · NCT04554940 · Dwarfism

Sources and methodology

  • Publications: ctgov.study_references (PubMed PMIDs auto-attached by ClinicalTrials.gov to each trial), reference types RESULT, DERIVED, and BACKGROUND.
  • Per-arm outcome values: ctgov.outcome_measurements joined to ctgov.design_outcomes where outcome_type = 'PRIMARY', restricted to phase PHASE3 and PHASE2/PHASE3.
  • Publication metadata (title, journal, year, authors): PubMed E-utilities efetch.fcgi
  • Data freshness: Tue, 26 May 2026 18:08:36 GMT.
This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).