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Study · Observational study · n = 35 · 2026

11 results

Real-world growth outcomes and safety of vosoritide in children with achondroplasia: a Brazilian single center 24 monhts experience.

Bordallo APN, Dominguez Alfonso JG, Llerena J Jr, Magalhaes TSPC, Botti H, Dain Gandelman Horovitz D, Collett-Solberg PF

Hormone research in paediatrics21 Jul 2026

PMID42479670 (opens in a new tab)DOI10.1159/000553585 (opens in a new tab)PepBankPUB-42479670
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 study11

11 results from studies that gave the peptide. 11 results from people.

  1. 01HumanObservational studyEfficacy outcome

    In children with achondroplasia receiving vosoritide, height velocity increased by +1.50 SDS at 12 months versus baseline (p < 0.001).

    In plain wordsIn children with achondroplasia, vosoritide treatment was linked to higher height velocity at 12 months than baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseHeight velocity at twelve months
    Study

    n = 35

    Comparator
    Baseline
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated · 12 monthsAs given in this study (human).

    Measurements

    Height velocity change vs baseline at 12 months
    1.5 SDS
    vs baseline

    Source sentences

    Summary
    Height velocity increased significantly at both 12 months (+1.50 SDS) and 24 months (+1.91 SDS) compared with baseline (p < 0.001).
    3 context sentences
    Summary · context
    We conducted a hybrid retrospective-prospective observational study in children with achondroplasia treated with vosoritide at a single tertiary center.
    Summary · context
    Prospectively patients were evaluated at baseline and after 12 and 24 months on treatment.
    Summary · context
    Thirty-five children (mean age 5.92 ± 3.56 years) were included.
  2. 02HumanObservational studyEfficacy outcome

    In children with achondroplasia receiving vosoritide, height velocity increased by +1.91 SDS at 24 months versus baseline (p < 0.001).

    In plain wordsIn children with achondroplasia, vosoritide treatment was linked to higher height velocity at 24 months than baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseHeight velocity at twenty-four months
    Study

    n = 35

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

    Measurements

    Height velocity change vs baseline at 24 months
    1.91 SDS
    vs baseline

    Source sentences

    Summary
    Height velocity increased significantly at both 12 months (+1.50 SDS) and 24 months (+1.91 SDS) compared with baseline (p < 0.001).
    3 context sentences
    Summary · context
    We conducted a hybrid retrospective-prospective observational study in children with achondroplasia treated with vosoritide at a single tertiary center.
    Summary · context
    Prospectively patients were evaluated at baseline and after 12 and 24 months on treatment.
    Summary · context
    Thirty-five children (mean age 5.92 ± 3.56 years) were included.
  3. 03HumanObservational studyEfficacy outcome

    In vosoritide-treated children with achondroplasia, achondroplasia-reference height SDS showed a trend toward increase at 12 months versus baseline (+0.17 SDS, p = 0.060).

    In plain wordsIn vosoritide-treated children with achondroplasia, achondroplasia-reference height scores showed a trend toward an increase at 12 months compared with baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseAchondroplasia-reference height SDS at twelve months
    Study

    n = 35

    Comparator
    Baseline
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated · 12 monthsAs given in this study (human).

    Measurements

    Achondroplasia-reference height SDS change vs baseline at 12 months
    0.17 SDS
    vs baseline; reported p = 0.060

    Source sentences

    Summary
    Using achondroplasia-specific references, height SDS increased over time, with a trend toward increment at 12 months (+0.17 SDS, p = 0.060) and a significant increase at 24 months (+0.50 SDS, p < 0.001).
    3 context sentences
    Summary · context
    We conducted a hybrid retrospective-prospective observational study in children with achondroplasia treated with vosoritide at a single tertiary center.
    Summary · context
    Prospectively patients were evaluated at baseline and after 12 and 24 months on treatment.
    Summary · context
    Thirty-five children (mean age 5.92 ± 3.56 years) were included.
  4. 04HumanObservational studyEfficacy outcome

    In vosoritide-treated children, achondroplasia-reference height SDS increased by +0.50 at 24 months versus baseline (p < 0.001).

    In plain wordsIn vosoritide-treated children, achondroplasia-reference height scores increased at 24 months compared with baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseAchondroplasia-reference height SDS at twenty-four months
    Study

    n = 35

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

    Measurements

    Achondroplasia-reference height SDS change vs baseline at 24 months
    0.5 SDS
    vs baseline

    Source sentences

    Summary
    Using achondroplasia-specific references, height SDS increased over time, with a trend toward increment at 12 months (+0.17 SDS, p = 0.060) and a significant increase at 24 months (+0.50 SDS, p < 0.001).
    3 context sentences
    Summary · context
    We conducted a hybrid retrospective-prospective observational study in children with achondroplasia treated with vosoritide at a single tertiary center.
    Summary · context
    Prospectively patients were evaluated at baseline and after 12 and 24 months on treatment.
    Summary · context
    Thirty-five children (mean age 5.92 ± 3.56 years) were included.
  5. 05HumanObservational studyEfficacy outcome

    In vosoritide-treated children with achondroplasia, WHO-based height SDS showed no significant change at 12 months versus baseline.

    In plain wordsIn vosoritide-treated children with achondroplasia, WHO-based height scores did not change significantly at 12 months compared with baseline.

    Peptide
    Vosoritide
    Outcome
    No significant changeWHO-reference height SDS at twelve months
    Study

    n = 35

    Comparator
    Baseline
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated · 12 monthsAs given in this study (human).

    Source sentences

    Summary
    Similarly, WHO-based height SDS showed no significant change at 12 months and a significant increment at 24 months (+0.30 SDS, p = 0.005), suggesting a more gradual shift toward WHO standards.
    3 context sentences
    Summary · context
    We conducted a hybrid retrospective-prospective observational study in children with achondroplasia treated with vosoritide at a single tertiary center.
    Summary · context
    Prospectively patients were evaluated at baseline and after 12 and 24 months on treatment.
    Summary · context
    Thirty-five children (mean age 5.92 ± 3.56 years) were included.
  6. 06HumanObservational studyEfficacy outcome

    In vosoritide-treated children with achondroplasia, WHO-based height SDS increased by +0.30 at 24 months versus baseline (p = 0.005).

    In plain wordsIn vosoritide-treated children with achondroplasia, WHO-based height scores increased at 24 months compared with baseline.

    Peptide
    Vosoritide
    Outcome
    IncreaseWHO-reference height SDS at twenty-four months
    Study

    n = 35

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

    Measurements

    WHO-reference height SDS change vs baseline at 24 months
    0.3 SDS
    vs baseline

    Source sentences

    Summary
    Similarly, WHO-based height SDS showed no significant change at 12 months and a significant increment at 24 months (+0.30 SDS, p = 0.005), suggesting a more gradual shift toward WHO standards.
    3 context sentences
    Summary · context
    We conducted a hybrid retrospective-prospective observational study in children with achondroplasia treated with vosoritide at a single tertiary center.
    Summary · context
    Prospectively patients were evaluated at baseline and after 12 and 24 months on treatment.
    Summary · context
    Thirty-five children (mean age 5.92 ± 3.56 years) were included.
  7. 07HumanObservational studyEfficacy outcome

    In children with achondroplasia receiving vosoritide, body proportionality remained unchanged versus baseline (p = 0.134).

    In plain wordsIn children with achondroplasia, body proportionality remained unchanged during vosoritide treatment compared with baseline.

    Peptide
    Vosoritide
    Outcome
    No significant changeBody proportionality
    Study

    n = 35

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

    Source sentences

    Summary
    Body proportionality remained unchanged (p = 0.134).
    3 context sentences
    Summary · context
    We conducted a hybrid retrospective-prospective observational study in children with achondroplasia treated with vosoritide at a single tertiary center.
    Summary · context
    Prospectively patients were evaluated at baseline and after 12 and 24 months on treatment.
    Summary · context
    Thirty-five children (mean age 5.92 ± 3.56 years) were included.
  8. 08HumanObservational studyEfficacy outcome

    In vosoritide-treated children, height velocity increment during the first 12 months did not significantly predict height SDS gain at 24 months.

    In plain wordsIn vosoritide-treated children, early height velocity change did not significantly predict later height score gains.

    Peptide
    Vosoritide
    Outcome
    No significant changeEarly height velocity as predictor of later height gain
    Study

    n = 35

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

    Source sentences

    Summary
    Height velocity increment during the first 12 months did not significantly predict height SDS gain at 24 months, highlighting inter-individual variability in treatment response.
    3 context sentences
    Summary · context
    We conducted a hybrid retrospective-prospective observational study in children with achondroplasia treated with vosoritide at a single tertiary center.
    Summary · context
    Prospectively patients were evaluated at baseline and after 12 and 24 months on treatment.
    Summary · context
    Thirty-five children (mean age 5.92 ± 3.56 years) were included.
  9. 09HumanObservational studyEfficacy outcome

    In children with achondroplasia receiving vosoritide, growth response showed a significant age-by-time interaction (p = 0.006).

    In plain wordsIn vosoritide-treated children with achondroplasia, growth response over time was suggested to differ by age at treatment start.

    Peptide
    Vosoritide
    Outcome
    Not applicableAge-by-time interaction in growth response
    Study

    n = 35

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

    Source sentences

    Summary
    A significant age-by-time interaction was observed (p = 0.006), suggesting a differential longitudinal response according to age at treatment initiation.
    3 context sentences
    Summary · context
    We conducted a hybrid retrospective-prospective observational study in children with achondroplasia treated with vosoritide at a single tertiary center.
    Summary · context
    Prospectively patients were evaluated at baseline and after 12 and 24 months on treatment.
    Summary · context
    Thirty-five children (mean age 5.92 ± 3.56 years) were included.
  10. 10HumanObservational studySafety outcome

    In children with achondroplasia receiving vosoritide, hypertrichosis occurred in 66% of patients.

    In plain wordsIn children with achondroplasia receiving vosoritide, excess hair growth occurred in 66% of patients.

    Peptide
    Vosoritide
    Outcome
    Not applicableHypertrichosis
    Study

    n = 35

    Outcome group
    Skin and hair · Skin and wound
    Studied dose
    Dose not stated

    Measurements

    Hypertrichosis
    66%
    patients

    Source sentences

    Summary
    Hypertrichosis occurred in 66% of patients, but no severe adverse events were reported.
    3 context sentences
    Summary · context
    We conducted a hybrid retrospective-prospective observational study in children with achondroplasia treated with vosoritide at a single tertiary center.
    Summary · context
    Prospectively patients were evaluated at baseline and after 12 and 24 months on treatment.
    Summary · context
    Thirty-five children (mean age 5.92 ± 3.56 years) were included.
  11. 11HumanObservational studySafety outcome

    In children with achondroplasia receiving vosoritide, no severe adverse events were reported.

    In plain wordsIn children with achondroplasia receiving vosoritide, no severe adverse events were reported.

    Peptide
    Vosoritide
    Outcome
    Not applicableSevere adverse events
    Study

    n = 35

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

    Source sentences

    Summary
    Hypertrichosis occurred in 66% of patients, but no severe adverse events were reported.
    3 context sentences
    Summary · context
    We conducted a hybrid retrospective-prospective observational study in children with achondroplasia treated with vosoritide at a single tertiary center.
    Summary · context
    Prospectively patients were evaluated at baseline and after 12 and 24 months on treatment.
    Summary · context
    Thirty-five children (mean age 5.92 ± 3.56 years) were included.

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.

Publication types

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