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Study · Case report or series · n = 1 · 2025

5 results

Functional Change in a Child With Achondroplasia Following 12 Months Treatment With Vosoritide-A Case Report.

Ireland P, Carroll T, Pendlebury E, Weston E, Munns C

American journal of medical genetics. Part A18 Mar 2025Cited by 3 (Europe PMC)

PMID40099812 (opens in a new tab)DOI10.1002/ajmg.a.64054 (opens in a new tab)PepBankPUB-40099812
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 seriesEfficacy outcome

    The boy receiving vosoritide had slightly higher independence than age-matched peers with achondroplasia.

    In plain wordsThe boy receiving vosoritide had slightly higher independence than age-matched peers with achondroplasia.

    Peptide
    Vosoritide
    Outcome
    IncreaseIndependence level
    Study

    n = 1

    Comparator
    Age-matched peers with achondroplasia
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    Dose not stated · injection · 12 monthsAs given in this study (human).

    Source sentences

    Summary
    He was found to have a slightly higher level of independence when compared to age-matched peers with achondroplasia,
    2 context sentences
    Summary · context
    Here, we present a 5-year-old boy with achondroplasia who received vosoritide injections for 12 months.
    Summary · context
    His functional performance was measured using the Functional Independence Measure for Children (WeeFIM).
  2. 02HumanCase report or seriesEfficacy outcome

    The vosoritide-treated boy improved independence skills twice as fast as children with achondroplasia following 12 months on vosoritide in a previous study.

    In plain wordsThe vosoritide-treated boy improved independence skills twice as fast as children with achondroplasia after 12 months on vosoritide in a previous study.

    Peptide
    Vosoritide
    Outcome
    IncreaseIndependence skill improvement rate
    Study

    n = 1

    Comparator
    Children with achondroplasia following 12 months on vosoritide in a previous study
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    Dose not stated · injection · 12 monthsAs given in this study (human).

    Source sentences

    Summary
    and his rate of improvement in independence skills was twice that recorded in a previous study monitoring independence in children with achondroplasia following 12 months on vosoritide.
    2 context sentences
    Summary · context
    Here, we present a 5-year-old boy with achondroplasia who received vosoritide injections for 12 months.
    Summary · context
    His functional performance was measured using the Functional Independence Measure for Children (WeeFIM).
  3. 03HumanCase report or seriesEfficacy outcome

    The boy receiving vosoritide had almost three times the total WeeFIM score change seen in average-statured normative data.

    In plain wordsThe boy receiving vosoritide showed almost three times the total independence score change in average-statured normative data.

    Peptide
    Vosoritide
    Outcome
    IncreaseTotal WeeFIM score change
    Study

    n = 1

    Comparator
    Average-statured normative data from ages 4 to 5 years
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    Dose not stated · injection · 12 monthsAs given in this study (human).

    Measurements

    Total WeeFIM change versus average-statured normative data
    ≈ 3 fold
    12 months, ages 4 to 5 years; source says almost three times

    Source sentences

    Summary
    When compared with WeeFIM normative data from average-statured children across the 12 months from 4 to 5 years, our child demonstrated almost three times the change for total WeeFIM and Self-Care domain scores and nine times the change across the Mobility domain scores, indicating a rapid increase i…
    2 context sentences
    Summary · context
    Here, we present a 5-year-old boy with achondroplasia who received vosoritide injections for 12 months.
    Summary · context
    His functional performance was measured using the Functional Independence Measure for Children (WeeFIM).
  4. 04HumanCase report or seriesEfficacy outcome

    The boy receiving vosoritide had almost three times the Self-Care score change seen in average-statured normative data.

    In plain wordsThe boy receiving vosoritide showed almost three times the self-care score change in average-statured normative data.

    Peptide
    Vosoritide
    Outcome
    IncreaseSelf-Care domain score change
    Study

    n = 1

    Comparator
    Average-statured normative data from ages 4 to 5 years
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    Dose not stated · injection · 12 monthsAs given in this study (human).

    Measurements

    Self-Care domain change versus average-statured normative data
    ≈ 3 fold
    12 months, ages 4 to 5 years; source says almost three times

    Source sentences

    Summary
    When compared with WeeFIM normative data from average-statured children across the 12 months from 4 to 5 years, our child demonstrated almost three times the change for total WeeFIM and Self-Care domain scores and nine times the change across the Mobility domain scores, indicating a rapid increase i…
    2 context sentences
    Summary · context
    Here, we present a 5-year-old boy with achondroplasia who received vosoritide injections for 12 months.
    Summary · context
    His functional performance was measured using the Functional Independence Measure for Children (WeeFIM).
  5. 05HumanCase report or seriesEfficacy outcome

    The boy receiving vosoritide had nine times the Mobility score change seen in average-statured normative data.

    In plain wordsThe boy receiving vosoritide showed nine times the mobility score change in average-statured normative data.

    Peptide
    Vosoritide
    Outcome
    IncreaseMobility domain score change
    Study

    n = 1

    Comparator
    Average-statured normative data from ages 4 to 5 years
    Outcome group
    Exercise and physical function · Musculoskeletal
    Studied dose
    Dose not stated · injection · 12 monthsAs given in this study (human).

    Measurements

    Mobility domain change versus average-statured normative data
    9 fold
    12 months, ages 4 to 5 years

    Source sentences

    Summary
    When compared with WeeFIM normative data from average-statured children across the 12 months from 4 to 5 years, our child demonstrated almost three times the change for total WeeFIM and Self-Care domain scores and nine times the change across the Mobility domain scores, indicating a rapid increase i…
    2 context sentences
    Summary · context
    Here, we present a 5-year-old boy with achondroplasia who received vosoritide injections for 12 months.
    Summary · context
    His functional performance was measured using the Functional Independence Measure for Children (WeeFIM).

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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