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Study · Observational study · n = 21 · 2026

6 results

Perceived Impact of Vosoritide on Health-Related Quality of Life in Children with Selected Genetic Causes of Short Stature: A Qualitative Study of Children and Caregivers.

Seaforth R, Shafaei N, Pitner K, Cho M, Romero B, de la Motte A, Brewer K, Kanakatti Shankar R, Dauber A

Hormone research in paediatrics4 Sept 2026

PMID42696489 (opens in a new tab)DOI10.1159/hrp/adaag004 (opens in a new tab)PepBankPUB-42696489
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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 study6

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

  1. 01HumanObservational studyEfficacy outcome

    After vosoritide initiation, 74% of caregivers reported changes in children's daily activities.

    In plain wordsAfter vosoritide initiation, 74% of caregivers reported changes in children's daily activities.

    Peptide
    Vosoritide
    Outcome
    Direction not reportedDaily activity changes reported by caregivers
    Study

    n = 21

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated · >2 yearsAs given in this study (human).

    Measurements

    Caregivers reporting daily activity changes
    74%

    Source sentences

    Summary
    After starting vosoritide, changes were most reported for impacts on daily activities by both caregiver (74%) and child (100%) participants.
    3 context sentences
    Summary · context
    We performed a cross-sectional, remote, qualitative interview study targeting children receiving vosoritide for >2 years as part of a clinical trial and their caregivers.
    Summary · context
    Twenty-one caregivers and 14 child participants from 20 families were interviewed.
    Summary · context
    Four conditions were represented in the sample (12 hypochondroplasia, 4 NPR2 mutations, 2 ACAN mutations, 2 Noonan syndrome).
  2. 02HumanObservational studyEfficacy outcome

    After vosoritide initiation, 12 caregivers reported improved ability to reach things in children with genetic short stature.

    In plain wordsAfter vosoritide initiation, 12 caregivers reported that children could reach things more easily.

    Peptide
    Vosoritide
    Outcome
    IncreaseAbility to reach things reported by caregivers
    Study

    n = 21

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated · >2 yearsAs given in this study (human).

    Measurements

    Caregivers reporting improved ability to reach things
    12 caregivers
    caregivers

    Source sentences

    Summary
    Most reported were "improved ability to reach things" (12 caregivers and 14 children) and "being more independent/needing less assistance" (9 caregivers and 5 children).
    3 context sentences
    Summary · context
    We performed a cross-sectional, remote, qualitative interview study targeting children receiving vosoritide for >2 years as part of a clinical trial and their caregivers.
    Summary · context
    Twenty-one caregivers and 14 child participants from 20 families were interviewed.
    Summary · context
    Four conditions were represented in the sample (12 hypochondroplasia, 4 NPR2 mutations, 2 ACAN mutations, 2 Noonan syndrome).
  3. 03HumanObservational studyEfficacy outcome

    After vosoritide initiation, 9 caregivers reported greater independence or less need for assistance in children.

    In plain wordsAfter vosoritide initiation, 9 caregivers reported that children were more independent or needed less assistance.

    Peptide
    Vosoritide
    Outcome
    IncreaseIndependence reported by caregivers
    Study

    n = 21

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated · >2 yearsAs given in this study (human).

    Measurements

    Caregivers reporting being more independent/needing less assistance
    9 caregivers
    caregivers

    Source sentences

    Summary
    Most reported were "improved ability to reach things" (12 caregivers and 14 children) and "being more independent/needing less assistance" (9 caregivers and 5 children).
    3 context sentences
    Summary · context
    We performed a cross-sectional, remote, qualitative interview study targeting children receiving vosoritide for >2 years as part of a clinical trial and their caregivers.
    Summary · context
    Twenty-one caregivers and 14 child participants from 20 families were interviewed.
    Summary · context
    Four conditions were represented in the sample (12 hypochondroplasia, 4 NPR2 mutations, 2 ACAN mutations, 2 Noonan syndrome).
  4. 04HumanObservational studyEfficacy outcome

    After vosoritide initiation, 100% of child participants reported changes in daily activities.

    In plain wordsAfter vosoritide initiation, 100% of child participants reported changes in daily activities.

    Peptide
    Vosoritide
    Outcome
    Direction not reportedDaily activity changes reported by children
    Study

    n = 14

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated · >2 yearsAs given in this study (human).

    Measurements

    Children reporting daily activity changes
    100%

    Source sentences

    Summary
    After starting vosoritide, changes were most reported for impacts on daily activities by both caregiver (74%) and child (100%) participants.
    3 context sentences
    Summary · context
    We performed a cross-sectional, remote, qualitative interview study targeting children receiving vosoritide for >2 years as part of a clinical trial and their caregivers.
    Summary · context
    Twenty-one caregivers and 14 child participants from 20 families were interviewed.
    Summary · context
    Four conditions were represented in the sample (12 hypochondroplasia, 4 NPR2 mutations, 2 ACAN mutations, 2 Noonan syndrome).
  5. 05HumanObservational studyEfficacy outcome

    After vosoritide initiation, 14 children reported improved ability to reach things.

    In plain wordsAfter vosoritide initiation, 14 children reported that they could reach things more easily.

    Peptide
    Vosoritide
    Outcome
    IncreaseAbility to reach things reported by children
    Study

    n = 14

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated · >2 yearsAs given in this study (human).

    Measurements

    Children reporting improved ability to reach things
    14 children
    children

    Source sentences

    Summary
    Most reported were "improved ability to reach things" (12 caregivers and 14 children) and "being more independent/needing less assistance" (9 caregivers and 5 children).
    3 context sentences
    Summary · context
    We performed a cross-sectional, remote, qualitative interview study targeting children receiving vosoritide for >2 years as part of a clinical trial and their caregivers.
    Summary · context
    Twenty-one caregivers and 14 child participants from 20 families were interviewed.
    Summary · context
    Four conditions were represented in the sample (12 hypochondroplasia, 4 NPR2 mutations, 2 ACAN mutations, 2 Noonan syndrome).
  6. 06HumanObservational studyEfficacy outcome

    After vosoritide initiation, 5 children reported greater independence or less need for assistance.

    In plain wordsAfter vosoritide initiation, 5 children reported that they were more independent or needed less assistance.

    Peptide
    Vosoritide
    Outcome
    IncreaseIndependence reported by children
    Study

    n = 14

    Outcome group
    Quality of life · Whole body
    Studied dose
    Dose not stated · >2 yearsAs given in this study (human).

    Measurements

    Children reporting being more independent/needing less assistance
    5 children
    children

    Source sentences

    Summary
    Most reported were "improved ability to reach things" (12 caregivers and 14 children) and "being more independent/needing less assistance" (9 caregivers and 5 children).
    3 context sentences
    Summary · context
    We performed a cross-sectional, remote, qualitative interview study targeting children receiving vosoritide for >2 years as part of a clinical trial and their caregivers.
    Summary · context
    Twenty-one caregivers and 14 child participants from 20 families were interviewed.
    Summary · context
    Four conditions were represented in the sample (12 hypochondroplasia, 4 NPR2 mutations, 2 ACAN mutations, 2 Noonan syndrome).

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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