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Study · Case report or series · n = 2 · 2026

5 results

Early Neonatal Administration of Vosoritide in Achondroplasia: A Report of Two Cases.

Sano S, Murai Y, Shimizu K, Nagai Y, Ishizaki R, Honda Y

American journal of medical genetics. Part A11 Mar 2026

PMID41810698 (opens in a new tab)DOI10.1002/ajmg.a.70116 (opens in a new tab)PepBankPUB-41810698
Publisher pagePubMedAsk about this study
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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study5

5 results from studies that gave the peptide. 5 results from people.

  1. 01HumanCase report or seriesSafety outcome

    Two infants with achondroplasia tolerated neonatal vosoritide without serious adverse events during short-term monitoring.

    In plain wordsTwo infants with achondroplasia had no serious adverse events during short-term vosoritide monitoring.

    Peptide
    Vosoritide
    Outcome
    Not applicableSerious adverse events
    Study

    n = 2

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Both patients tolerated treatment in the short term without serious adverse events during inpatient or outpatient monitoring.
    1 context sentence
    Summary · context
    We report two infants with genetically confirmed ACH who received daily subcutaneous vosoritide starting on postnatal days 8 and 9, representing the earliest initiation reported to date.
  2. 02HumanCase report or seriesEfficacy outcome

    Both infants with achondroplasia developed progressive foramen magnum stenosis during infancy despite neonatal vosoritide initiation.

    In plain wordsBoth infants developed progressive narrowing of the skull opening during vosoritide treatment.

    Peptide
    Vosoritide
    Outcome
    IncreaseForamen magnum stenosis progression
    Study

    n = 2

    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Despite early initiation, serial magnetic resonance imaging demonstrated progressive foramen magnum stenosis during infancy, and both patients required decompression surgery at 9 and 5 months of age, respectively,
    1 context sentence
    Summary · context
    We report two infants with genetically confirmed ACH who received daily subcutaneous vosoritide starting on postnatal days 8 and 9, representing the earliest initiation reported to date.
  3. 03HumanCase report or seriesOther

    Both vosoritide-treated infants required decompression surgery at 9 and 5 months of age, respectively.

    In plain wordsBoth vosoritide-treated infants required decompression surgery during infancy.

    Peptide
    Vosoritide
    Outcome
    Not applicableDecompression surgery
    Study

    n = 2

    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    both patients required decompression surgery at 9 and 5 months of age, respectively,
    1 context sentence
    Summary · context
    We report two infants with genetically confirmed ACH who received daily subcutaneous vosoritide starting on postnatal days 8 and 9, representing the earliest initiation reported to date.
  4. 04HumanCase report or seriesEfficacy outcome

    In two vosoritide-treated infants, growth velocity trajectories were generally above achondroplasia-specific reference medians at comparable ages.

    In plain wordsIn two vosoritide-treated infants, growth velocity was generally above reference medians for infants with achondroplasia.

    Peptide
    Vosoritide
    Outcome
    IncreaseGrowth velocity versus reference medians
    Study

    n = 2

    Comparator
    Achondroplasia-specific reference medians at comparable ages
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Growth trajectories, assessed by growth velocity (GV), were generally above the ACH-specific reference medians at comparable ages, while the characteristic slowing of GV during infancy was preserved.
    1 context sentence
    Summary · context
    We report two infants with genetically confirmed ACH who received daily subcutaneous vosoritide starting on postnatal days 8 and 9, representing the earliest initiation reported to date.
  5. 05HumanCase report or seriesEfficacy outcome

    In two vosoritide-treated infants, the characteristic slowing of growth velocity during infancy remained present.

    In plain wordsIn two vosoritide-treated infants, growth velocity still slowed during infancy.

    Peptide
    Vosoritide
    Outcome
    DecreaseInfant growth velocity slowing
    Study

    n = 2

    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Growth trajectories, assessed by growth velocity (GV), were generally above the ACH-specific reference medians at comparable ages, while the characteristic slowing of GV during infancy was preserved.
    1 context sentence
    Summary · context
    We report two infants with genetically confirmed ACH who received daily subcutaneous vosoritide starting on postnatal days 8 and 9, representing the earliest initiation reported to date.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    28 Oct 2026, 02:03 UTC

    Study results updated

  2. Version 1

    27 Oct 2026, 16:37 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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