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Study · Case report or series · n = 1 · 2026

2 results

Early initiation of therapy with vosoritide: case report. Description of the first Italian patient affected by achondroplasia treated with vosoritide before 2 years of age.

Kalapurackal MA, Wanker P

Frontiers in pediatrics6 Jul 2026

PMID42519733 (opens in a new tab)PMCIDPMC13381835 (opens in a new tab)DOI10.3389/fped.2026.1741018 (opens in a new tab)PepBankPUB-42519733
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Summary

Achondroplasia, the most common skeletal dysplasia, is mainly characterized by disproportionate short stature. Affected patients present a reduced linear growth, and the greatest decrease in growth velocity is evident in infancy. Vosoritide is the first approved drug to improve linear growth in children affected by achondroplasia. Since 2023, it is approved in children from birth by the FDA, and from 4 months of age by the EMA. However, until October 2025, the prescription and reimbursement of vosoritide in Italy was only possible starting from 2 years of age. We present the case of the first patient affected by achondroplasia treated with vosoritide in Italy before 2 years of age, managed thanks to the approval by the local rare diseases commission. Therapy with vosoritide was initiated at 7 months of age. The infant presented no severe adverse effects. She completed 12 months of therapy, at which time an improvement of +0.35 SD in length, according to growth curves specific for girls affected by achondroplasia, was documented. This result is in line with previous studies and underlines the importance of an early therapy initiation.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study2

2 results from studies that gave the peptide. 2 results from people.

  1. 01HumanCase report or seriesSafety outcome

    The infant receiving vosoritide had no severe adverse effects.

    In plain wordsThe infant receiving vosoritide had no severe adverse effects.

    Peptide
    Vosoritide
    Outcome
    Not applicableSevere adverse effects
    Study

    n = 1

    Outcome group
    Adverse events · Whole body
    Studied dose
    Dose not stated · 12 monthsAs given in this study (human).

    Source sentences

    Summary
    The infant presented no severe adverse effects.
    2 context sentences
    Summary · context
    We present the case of the first patient affected by achondroplasia treated with vosoritide in Italy before 2 years of age, managed thanks to the approval by the local rare diseases commission.
    Summary · context
    Therapy with vosoritide was initiated at 7 months of age.
  2. 02HumanCase report or seriesEfficacy outcome

    The infant's length improved by +0.35 SD after 12 months of vosoritide, using achondroplasia-specific growth curves.

    In plain wordsThe infant's length score improved by +0.35 SD after 12 months of vosoritide.

    Peptide
    Vosoritide
    Outcome
    IncreaseLength standard deviation score
    Study

    n = 1

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

    Measurements

    Length improvement
    0.35 SD
    achondroplasia-specific curves

    Source sentences

    Summary
    She completed 12 months of therapy, at which time an improvement of +0.35 SD in length, according to growth curves specific for girls affected by achondroplasia, was documented.
    2 context sentences
    Summary · context
    We present the case of the first patient affected by achondroplasia treated with vosoritide in Italy before 2 years of age, managed thanks to the approval by the local rare diseases commission.
    Summary · context
    Therapy with vosoritide was initiated at 7 months of age.

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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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