Real-World Evidence on the Effectiveness and Safety of Vosoritide in Latin American Patients With Achondroplasia (EVOLAC).

De Victor J, Gil ED, Pabletich F, Moleón RR, Dellamea CA, Fernández M, Serrano NC, Maradei S, Gordillo-González G, Rosselli P, De León Ojeda NE, De la Fuente-Cortez BE, Unanue N, Lacarrubba-Flores MD, Gueçaimburu R

American journal of medical genetics. Part A31 Aug 2026

PMID42675811DOI10.1002/ajmg.a.70254PepBankPUB-42675811

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Summary

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Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study12

12 results from studies that gave the peptide. 12 results from people.

01HumanObservational studyOther
Children with achondroplasia receiving vosoritide spent 93.9% of treatment time without interruptions, with mean treatment duration of 539 days.
In plain wordsChildren with achondroplasia receiving vosoritide spent 93.9% of treatment time without interruptions.
Peptide
Vosoritide
Outcome
Not applicableTreatment adherence
Study
n = 52
Outcome group
Treatment adherence and retention · Whole body
Studied dose
Dose not stated · Mean 539 daysAs given in this study (human).
Measurements
Time without interruptions
93.9%
Source sentences
Summary
Fifty-two patients (mean age 5.9 years at treatment initiation) were included, with a mean treatment duration of 539 days and high adherence (93.9% time without interruptions).
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile.
Summary · context
Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581101
02HumanObservational studyEfficacy outcome
All children with achondroplasia receiving vosoritide had higher growth velocity percentiles during treatment than before treatment, sometimes exceeding the 95th percentile.
In plain wordsAll children with achondroplasia receiving vosoritide had higher growth velocity percentiles during treatment than before treatment.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity percentile
Study
n = 52
Comparator
Before treatment
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Measurements
Annualized growth velocity percentile exceeded in some cases
> 95 percentile
during treatment vs reference curves
Source sentences
Summary
All patients were in a higher percentile range for AGV during treatment compared to before treatment, in some cases exceeding the 95th percentile, when compared to reference curves.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile.
Summary · context
Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581104
03HumanObservational studyEfficacy outcome
Body proportion indices remained stable in children with achondroplasia receiving vosoritide.
In plain wordsBody proportions remained stable in children with achondroplasia receiving vosoritide.
Peptide
Vosoritide
Outcome
No significant changeBody proportion indices
Study
n = 52
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Source sentences
Summary
Body proportion indices remained stable.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile.
Summary · context
Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581105
04HumanObservational studyEfficacy outcome
All children with achondroplasia receiving vosoritide gained linear growth, with variation in the magnitude between individuals.
In plain wordsAll children with achondroplasia receiving vosoritide gained linear growth, with different increases between children.
Peptide
Vosoritide
Outcome
IncreaseLinear growth
Study
n = 52
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Source sentences
Summary
All patients demonstrated gains in linear growth, although the magnitude of increase varied across individuals.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile.
Summary · context
Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581106
05HumanObservational studySafety outcome
Children with achondroplasia receiving vosoritide had frequent but mild adverse events, mainly injection-site reactions.
In plain wordsChildren with achondroplasia receiving vosoritide had frequent but mild adverse events, mainly injection-site reactions.
Peptide
Vosoritide
Outcome
Not applicableMild adverse events and injection-site reactions
Study
n = 52
Outcome group
Adverse events · Whole body
Studied dose
Dose not stated
Source sentences
Summary
Adverse events were frequent but mild, mainly injection-site reactions;
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile.
Summary · context
Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581107
06HumanObservational studySafety outcome
No severe adverse events occurred in children with achondroplasia receiving vosoritide.
In plain wordsNo severe adverse events occurred in children with achondroplasia receiving vosoritide.
Peptide
Vosoritide
Outcome
Not applicableSevere adverse events
Study
n = 52
Outcome group
Adverse events · Whole body
Studied dose
Dose not stated
Source sentences
Summary
Adverse events were frequent but mild, mainly injection-site reactions; no severe adverse events or treatment discontinuations due to safety issues occurred.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile.
Summary · context
Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581108
07HumanObservational studySafety outcome
No children receiving vosoritide discontinued treatment because of safety issues.
In plain wordsNo children receiving vosoritide discontinued treatment because of safety issues.
Peptide
Vosoritide
Outcome
Not applicableSafety-related treatment discontinuations
Study
n = 52
Outcome group
Adverse events · Whole body
Studied dose
Dose not stated
Source sentences
Summary
Adverse events were frequent but mild, mainly injection-site reactions; no severe adverse events or treatment discontinuations due to safety issues occurred.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile.
Summary · context
Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581109
08HumanObservational studyEfficacy outcome
Among caregivers of children receiving vosoritide, 98% perceived positive changes in overall well-being, daily functioning, or quality of life.
In plain wordsAmong caregivers of children receiving vosoritide, 98% perceived positive changes in well-being, daily functioning, or quality of life.
Peptide
Vosoritide
Outcome
IncreaseCaregiver-perceived benefit
Study
n = 52
Outcome group
Quality of life · Whole body
Studied dose
Dose not stated
Measurements
Caregivers perceiving benefit
98%
Source sentences
Summary
Parent-reported outcomes were highly favorable, with 98% of caregivers perceiving positive changes in their children's overall well-being, daily functioning, or quality of life, and all caregivers expressing willingness to recommend the treatment.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile.
Summary · context
Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581110
09HumanObservational studyOther
All caregivers of children receiving vosoritide expressed willingness to recommend treatment.
In plain wordsAll caregivers of children receiving vosoritide expressed willingness to recommend treatment.
Peptide
Vosoritide
Outcome
Not applicableCaregiver willingness to recommend treatment
Study
n = 52
Outcome group
Patient-reported outcomes · Whole body
Studied dose
Dose not stated
Source sentences
Summary
Parent-reported outcomes were highly favorable, with 98% of caregivers perceiving positive changes in their children's overall well-being, daily functioning, or quality of life, and all caregivers expressing willingness to recommend the treatment.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile.
Summary · context
Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581111
10HumanObservational studyEfficacy outcome
In children receiving vosoritide, growth increases were consistent across sexes and age groups.
In plain wordsIn children receiving vosoritide, growth increases were consistent across sexes and age groups.
Peptide
Vosoritide
Outcome
IncreaseGrowth increases across sex and age groups
Study
n = 52
Outcome group
Linear growth · Musculoskeletal
Studied dose
Dose not stated
Source sentences
Summary
Increases were consistent across sexes and age groups.
2 context sentences(design, population, supporting numbers)
Summary · context
Height Z-scores increased significantly from 6 to 36 months (0.33 to 1.58; p < 0.001 to p < 0.05). The most significant impact in AVG is observed during the first year of treatment with vosoritide, followed by a subsequent stabilization, reaching growth rates comparable to those of the healthy population.
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile. Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581112
11HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, height Z-scores increased significantly from 6 to 36 months (0.33 to 1.58).
In plain wordsIn children with achondroplasia receiving vosoritide, height scores increased significantly from 6 to 36 months.
Peptide
Vosoritide
Outcome
IncreaseHeight Z-score
Study
n: not reported in source
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Measurements
Height Z-score increase range
0.33 to 1.58 Z-score
6 to 36 months
p
< 0.001
reported range lower threshold
p
< 0.05
reported range upper threshold
Source sentences
Summary
Height Z-scores increased significantly from 6 to 36 months (0.33 to 1.58; p < 0.001 to p < 0.05).
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile.
Summary · context
Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581102
12HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, growth velocity showed its largest impact in the first year and then stabilized near healthy-population rates.
In plain wordsChildren with achondroplasia receiving vosoritide showed their largest growth velocity impact in the first year, followed by stabilization near healthy-population rates.
Peptide
Vosoritide
Outcome
MixedAnnualized growth velocity trajectory
Study
n: not reported in source
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Source sentences
Summary
The most significant impact in AVG is observed during the first year of treatment with vosoritide, followed by a subsequent stabilization, reaching growth rates comparable to those of the healthy population.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective, multicenter, multinational cohort study including children with molecularly confirmed achondroplasia treated with vosoritide in centers from Argentina, Colombia, Uruguay, Mexico, and Chile.
Summary · context
Eligible patients had ≥ 6 months of treatment and at least two anthropometric evaluations.
Report a problemFND-4267581103

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.

Version 2Current
28 Oct 2026, 02:03 UTC
Study results updated
Version 1
27 Oct 2026, 16:37 UTC
First added · source update from Europe PMC

Subjects

Terms as the source lists them.

Publication types

Journal Article

Keywords

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