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Study · Observational study · n = 27 · 2025

6 results

Real-World Safety and Effectiveness of Vosoritide in Achondroplasia: Results from a Single Center in Portugal.

Rua IB, Silva I, Beger C, Gomes C, Pais MJ, Mirante A, Sousa SB

Advances in therapy5 Jun 2025Cited by 6 (Europe PMC)

PMID40471380 (opens in a new tab)PMCIDPMC12313777 (opens in a new tab)DOI10.1007/s12325-025-03223-6 (opens in a new tab)PepBankPUB-40471380
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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: CC BY-NC. Results still quote short attributed sentences.

Results from this study6

6 results from studies that gave the peptide. 6 results from people.

  1. 01HumanObservational studySafety outcome

    In children with achondroplasia receiving vosoritide, injection site reactions were the most common adverse drug reaction, reported in 14 children.

    In plain wordsIn children receiving vosoritide, injection site reactions were the most common adverse drug reaction.

    Peptide
    Vosoritide
    Outcome
    Not applicableInjection site reactions
    Study

    n = 27

    Outcome group
    Adverse events · Skin and wound
    Studied dose
    Dose not stated

    Measurements

    Children with injection site reactions
    14 children
    most common adverse drug reaction

    Source sentences

    Summary
    Injection site reactions were the most common adverse drug reaction observed (n = 14); no serious adverse drug reactions were reported.
    2 context sentences
    Summary · context
    This retrospective cohort study evaluated growth, safety, and treatment compliance in children with achondroplasia receiving vosoritide under an early access program in Portugal.
    Summary · context
    Twenty-seven children aged 2-14 years with a genetically confirmed diagnosis of achondroplasia were treated with vosoritide at a single Portuguese center for at least 6 months between January 2022 and June 2024.
  2. 02HumanObservational studySafety outcome

    In children with achondroplasia receiving vosoritide, no serious adverse drug reactions were reported.

    In plain wordsNo serious adverse drug reactions were reported in children receiving vosoritide.

    Peptide
    Vosoritide
    Outcome
    Not applicableSerious adverse drug reactions
    Study

    n = 27

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    Injection site reactions were the most common adverse drug reaction observed (n = 14); no serious adverse drug reactions were reported.
    2 context sentences
    Summary · context
    This retrospective cohort study evaluated growth, safety, and treatment compliance in children with achondroplasia receiving vosoritide under an early access program in Portugal.
    Summary · context
    Twenty-seven children aged 2-14 years with a genetically confirmed diagnosis of achondroplasia were treated with vosoritide at a single Portuguese center for at least 6 months between January 2022 and June 2024.
  3. 03HumanObservational studySafety outcome

    In children with achondroplasia receiving vosoritide, no discontinuations resulted from adverse drug reactions.

    In plain wordsNo children stopped vosoritide because of adverse drug reactions.

    Peptide
    Vosoritide
    Outcome
    Not applicableDiscontinuation due to adverse drug reactions
    Study

    n = 27

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated

    Source sentences

    Summary
    There were no discontinuations due to adverse drug reactions.
    2 context sentences
    Summary · context
    This retrospective cohort study evaluated growth, safety, and treatment compliance in children with achondroplasia receiving vosoritide under an early access program in Portugal.
    Summary · context
    Twenty-seven children aged 2-14 years with a genetically confirmed diagnosis of achondroplasia were treated with vosoritide at a single Portuguese center for at least 6 months between January 2022 and June 2024.
  4. 04HumanObservational studyEfficacy outcome

    In children receiving vosoritide, height SDS relative to an untreated achondroplasia population increased by 0.95 SD from baseline (P ≤ 0.0001).

    In plain wordsIn children receiving vosoritide, achondroplasia-specific height scores increased from baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseAchondroplasia-specific height SDS
    Study

    n = 15

    Comparator
    Baseline
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated · 24 monthsAs given in this study (human).

    Measurements

    Height SDS change versus untreated achondroplasia-specific population
    0.95 SD
    after 24 months versus baseline

    Source sentences

    Summary
    After 24 months of treatment, mean variation in height SDS increased from baseline by + 0.95 SD (P ≤ 0.0001), referenced to an untreated achondroplasia-specific population, and + 0.56 SD (P ≤ 0.0001) relative to children of average stature.
    2 context sentences
    Summary · context
    This retrospective cohort study evaluated growth, safety, and treatment compliance in children with achondroplasia receiving vosoritide under an early access program in Portugal.
    Summary · context
    Twenty-seven children aged 2-14 years with a genetically confirmed diagnosis of achondroplasia were treated with vosoritide at a single Portuguese center for at least 6 months between January 2022 and June 2024.
  5. 05HumanObservational studyEfficacy outcome

    In children receiving vosoritide, height SDS relative to average-stature children increased by 0.56 SD from baseline (P ≤ 0.0001).

    In plain wordsIn children receiving vosoritide, average-stature height scores increased from baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseAverage-stature height SDS
    Study

    n = 15

    Comparator
    Baseline
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated · 24 monthsAs given in this study (human).

    Measurements

    Height SDS change relative to children of average stature
    0.56 SD
    after 24 months versus baseline

    Source sentences

    Summary
    After 24 months of treatment, mean variation in height SDS increased from baseline by + 0.95 SD (P ≤ 0.0001), referenced to an untreated achondroplasia-specific population, and + 0.56 SD (P ≤ 0.0001) relative to children of average stature.
    2 context sentences
    Summary · context
    This retrospective cohort study evaluated growth, safety, and treatment compliance in children with achondroplasia receiving vosoritide under an early access program in Portugal.
    Summary · context
    Twenty-seven children aged 2-14 years with a genetically confirmed diagnosis of achondroplasia were treated with vosoritide at a single Portuguese center for at least 6 months between January 2022 and June 2024.
  6. 06HumanObservational studyEfficacy outcome

    In children with achondroplasia receiving vosoritide, mean AGV was 5.87 cm/year (95% CI 5.14-6.60), with an increase of 1.62 cm/year from baseline (P ≤ 0.0001).

    In plain wordsIn children receiving vosoritide, annualized growth velocity increased from baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseAnnualized growth velocity
    Study

    n: not reported in source

    Comparator
    Baseline
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated

    Measurements

    Mean annualized growth velocity
    5.87 cm/year
    from baseline
    95% CI of mean annualized growth velocity
    5.14 to 6.6 cm/year
    95% confidence interval
    Increase in annualized growth velocity
    1.62 cm/year
    increase from baseline

    Source sentences

    Summary
    Additionally, mean AGV from baseline was 5.87 cm/year (95% confidence interval 5.14-6.60), resulting in a significant increase of + 1.62 cm/year (P ≤ 0.0001).
    2 context sentences
    Summary · context
    This retrospective cohort study evaluated growth, safety, and treatment compliance in children with achondroplasia receiving vosoritide under an early access program in Portugal.
    Summary · context
    Twenty-seven children aged 2-14 years with a genetically confirmed diagnosis of achondroplasia were treated with vosoritide at a single Portuguese center for at least 6 months between January 2022 and June 2024.

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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