Retrospective study of foramen magnum development in patients with achondroplasia starting vosoritide before age 3.

Nakagawa M, Adachi N, Umekawa M, Hasegawa H, Okada K, Tanaka H

Genetics in medicine : official journal of the American College of Medical Genetics23 Jul 2026

PMID42495828DOI10.1016/j.gim.2026.102666PepBankPUB-42495828

Publisher page PubMedAsk about this study

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.

01HumanObservational studyEfficacy outcome
In vosoritide-treated children with achondroplasia, height SDS changes did not correlate significantly with foramen magnum diameter.
In plain wordsIn vosoritide-treated children, height score changes were not significantly linked to skull opening diameter.
Peptide
Vosoritide
Outcome
No significant changeHeight change and foramen magnum diameter correlation
Study
n = 9
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · Mean 2.5 yearsAs given in this study (human).
Source sentences
Summary
No significant correlation was identified between changes in body height standard deviation score and foramen magnum diameter.
3 context sentences(design, population, supporting numbers)
Summary · context
This retrospective, single-center case series included 9 patients who initiated vosoritide before age 3 years.
Summary · context
The anteroposterior and transverse diameters of the foramen magnum were measured and compared with reference curves from untreated ACH cohorts.
Summary · context
Vosoritide was initiated at a median age of 4 months, with a mean treatment duration of 2.5 years.
Report a problemFND-4249582806
02HumanCase report or seriesEfficacy outcome
In vosoritide-treated children with achondroplasia, 8 of 9 had steeper anteroposterior foramen magnum growth rates than untreated cohorts.
In plain wordsIn vosoritide-treated children with achondroplasia, most had faster anteroposterior skull opening growth than untreated cohorts.
Peptide
Vosoritide
Outcome
IncreaseAnteroposterior foramen magnum growth
Study
n = 9
Comparator
Untreated achondroplasia cohorts
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · Mean 2.5 yearsAs given in this study (human).
Measurements
Patients with steeper anteroposterior growth rate than untreated cohorts
8 patients
of 9 treated patients vs untreated reference cohorts
Source sentences
Summary
Compared with untreated cohorts, steeper growth rates were observed in 8 of the 9 patients for the anteroposterior diameter and in all patients for the transverse diameter.
3 context sentences(design, population, supporting numbers)
Summary · context
This retrospective, single-center case series included 9 patients who initiated vosoritide before age 3 years.
Summary · context
The anteroposterior and transverse diameters of the foramen magnum were measured and compared with reference curves from untreated ACH cohorts.
Summary · context
Vosoritide was initiated at a median age of 4 months, with a mean treatment duration of 2.5 years.
Report a problemFND-4249582801
03HumanCase report or seriesEfficacy outcome
All vosoritide-treated children with achondroplasia had steeper transverse foramen magnum growth rates than untreated cohorts.
In plain wordsAll vosoritide-treated children had faster transverse skull opening growth than untreated achondroplasia cohorts.
Peptide
Vosoritide
Outcome
IncreaseTransverse foramen magnum growth
Study
n = 9
Comparator
Untreated achondroplasia cohorts
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · Mean 2.5 yearsAs given in this study (human).
Source sentences
Summary
Compared with untreated cohorts, steeper growth rates were observed in 8 of the 9 patients for the anteroposterior diameter and in all patients for the transverse diameter.
3 context sentences(design, population, supporting numbers)
Summary · context
This retrospective, single-center case series included 9 patients who initiated vosoritide before age 3 years.
Summary · context
The anteroposterior and transverse diameters of the foramen magnum were measured and compared with reference curves from untreated ACH cohorts.
Summary · context
Vosoritide was initiated at a median age of 4 months, with a mean treatment duration of 2.5 years.
Report a problemFND-4249582802
04HumanCase report or seriesEfficacy outcome
Most vosoritide-treated children with achondroplasia remained at AFMS 1 or 2.
In plain wordsMost vosoritide-treated children with achondroplasia remained at skull opening stenosis scores of 1 or 2.
Peptide
Vosoritide
Outcome
Not applicableForamen magnum stenosis score
Study
n = 9
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · Mean 2.5 yearsAs given in this study (human).
Source sentences
Summary
Most patients remained at AFMS 1 or 2.
3 context sentences(design, population, supporting numbers)
Summary · context
This retrospective, single-center case series included 9 patients who initiated vosoritide before age 3 years.
Summary · context
The anteroposterior and transverse diameters of the foramen magnum were measured and compared with reference curves from untreated ACH cohorts.
Summary · context
Vosoritide was initiated at a median age of 4 months, with a mean treatment duration of 2.5 years.
Report a problemFND-4249582803
05HumanCase report or seriesEfficacy outcome
One vosoritide-treated child with achondroplasia progressed to AFMS 3.
In plain wordsOne vosoritide-treated child progressed to skull opening stenosis score 3.
Peptide
Vosoritide
Outcome
Not applicableProgression to AFMS three
Study
n = 9
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Measurements
Patients progressing to AFMS 3
1 patients
of 9 treated patients
Source sentences
Summary
One patient progressed to AFMS 3 and underwent decompression surgery at age 11 months.
3 context sentences(design, population, supporting numbers)
Summary · context
This retrospective, single-center case series included 9 patients who initiated vosoritide before age 3 years.
Summary · context
The anteroposterior and transverse diameters of the foramen magnum were measured and compared with reference curves from untreated ACH cohorts.
Summary · context
Vosoritide was initiated at a median age of 4 months, with a mean treatment duration of 2.5 years.
Report a problemFND-4249582804
06HumanCase report or seriesEfficacy outcome
One vosoritide-treated child with achondroplasia underwent decompression surgery at age 11 months.
In plain wordsOne vosoritide-treated child with achondroplasia underwent decompression surgery at age 11 months.
Peptide
Vosoritide
Outcome
Not applicableDecompression surgery
Study
n = 9
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Measurements
Patients undergoing decompression surgery
1 patients
of 9 treated patients
Age at decompression surgery
11 months
the one patient
Source sentences
Summary
One patient progressed to AFMS 3 and underwent decompression surgery at age 11 months.
3 context sentences(design, population, supporting numbers)
Summary · context
This retrospective, single-center case series included 9 patients who initiated vosoritide before age 3 years.
Summary · context
The anteroposterior and transverse diameters of the foramen magnum were measured and compared with reference curves from untreated ACH cohorts.
Summary · context
Vosoritide was initiated at a median age of 4 months, with a mean treatment duration of 2.5 years.
Report a problemFND-4249582805

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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.

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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