Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.

<p>Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.</p>

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Main Author: Francesco Muntoni (228210) (author)
Other Authors: James Signorovitch (672562) (author), Nathalie Goemans (503769) (author), Adnan Y. Manzur (7340012) (author), Nicolae Done (10751202) (author), Gautam Sajeev (6810248) (author), Jiayang Li (449346) (author), Hanane Akbarnejad (21615534) (author), Aarushi Sharma (5216615) (author), Susan J. Ward (3018144) (author), Erik H. Niks (674893) (author), Laurant Servais (21615537) (author), Eugenio Mercuri (289290) (author), Michela Guglieri (489528) (author), Volker Straub (251869) (author), Imelda de Groot (4399726) (author), Deborah Ridout (9096296) (author), Craig McDonald (2615428) (author)
Published: 2025
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author Francesco Muntoni (228210)
author2 James Signorovitch (672562)
Nathalie Goemans (503769)
Adnan Y. Manzur (7340012)
Nicolae Done (10751202)
Gautam Sajeev (6810248)
Jiayang Li (449346)
Hanane Akbarnejad (21615534)
Aarushi Sharma (5216615)
Susan J. Ward (3018144)
Erik H. Niks (674893)
Laurant Servais (21615537)
Eugenio Mercuri (289290)
Michela Guglieri (489528)
Volker Straub (251869)
Imelda de Groot (4399726)
Deborah Ridout (9096296)
Craig McDonald (2615428)
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author_facet Francesco Muntoni (228210)
James Signorovitch (672562)
Nathalie Goemans (503769)
Adnan Y. Manzur (7340012)
Nicolae Done (10751202)
Gautam Sajeev (6810248)
Jiayang Li (449346)
Hanane Akbarnejad (21615534)
Aarushi Sharma (5216615)
Susan J. Ward (3018144)
Erik H. Niks (674893)
Laurant Servais (21615537)
Eugenio Mercuri (289290)
Michela Guglieri (489528)
Volker Straub (251869)
Imelda de Groot (4399726)
Deborah Ridout (9096296)
Craig McDonald (2615428)
author_role author
dc.creator.none.fl_str_mv Francesco Muntoni (228210)
James Signorovitch (672562)
Nathalie Goemans (503769)
Adnan Y. Manzur (7340012)
Nicolae Done (10751202)
Gautam Sajeev (6810248)
Jiayang Li (449346)
Hanane Akbarnejad (21615534)
Aarushi Sharma (5216615)
Susan J. Ward (3018144)
Erik H. Niks (674893)
Laurant Servais (21615537)
Eugenio Mercuri (289290)
Michela Guglieri (489528)
Volker Straub (251869)
Imelda de Groot (4399726)
Deborah Ridout (9096296)
Craig McDonald (2615428)
dc.date.none.fl_str_mv 2025-06-27T17:40:29Z
dc.identifier.none.fl_str_mv 10.1371/journal.pone.0325736.s009
dc.relation.none.fl_str_mv https://figshare.com/articles/figure/Predicted_Change_in_Imputed_and_Unimputed_NSAA_Total_Score_Up_to_60_Months_Post-Baseline_Baseline_among_Patients_with_Baseline_Age_10_Years_/29427473
dc.rights.none.fl_str_mv CC BY 4.0
info:eu-repo/semantics/openAccess
dc.subject.none.fl_str_mv Medicine
Genetics
Environmental Sciences not elsewhere classified
Biological Sciences not elsewhere classified
Mathematical Sciences not elsewhere classified
Chemical Sciences not elsewhere classified
duchenne muscular dystrophy
designing randomized trials
69 %) relative
(> 18 months
body mass index
nsaa total score
ambulatory motor function
quartile range 1
dmd aged 4
div >< p
daily corticosteroid regimens
5 nsaa units
6 years ).
index year
dmd ).
initially ambulatory
6 years
5 years
16 years
uz leuven
steroid type
run velocity
receiving prednisone
receiving corticosteroids
prediction errors
novel therapies
modifying therapies
meter walk
mean age
longitudinal data
genotype class
floor velocity
drug development
dmd </
discuss applications
data source
cureduchenne ),
controlled studies
contextualizing outcomes
among n
ambulatory patients
31 %).
dc.title.none.fl_str_mv Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.
dc.type.none.fl_str_mv Image
Figure
info:eu-repo/semantics/publishedVersion
image
description <p>Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.</p>
eu_rights_str_mv openAccess
id Manara_a413adbbb6ee7c9c752523dd3c2d112e
identifier_str_mv 10.1371/journal.pone.0325736.s009
network_acronym_str Manara
network_name_str ManaraRepo
oai_identifier_str oai:figshare.com:article/29427473
publishDate 2025
repository.mail.fl_str_mv
repository.name.fl_str_mv
repository_id_str
rights_invalid_str_mv CC BY 4.0
spelling Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.Francesco Muntoni (228210)James Signorovitch (672562)Nathalie Goemans (503769)Adnan Y. Manzur (7340012)Nicolae Done (10751202)Gautam Sajeev (6810248)Jiayang Li (449346)Hanane Akbarnejad (21615534)Aarushi Sharma (5216615)Susan J. Ward (3018144)Erik H. Niks (674893)Laurant Servais (21615537)Eugenio Mercuri (289290)Michela Guglieri (489528)Volker Straub (251869)Imelda de Groot (4399726)Deborah Ridout (9096296)Craig McDonald (2615428)MedicineGeneticsEnvironmental Sciences not elsewhere classifiedBiological Sciences not elsewhere classifiedMathematical Sciences not elsewhere classifiedChemical Sciences not elsewhere classifiedduchenne muscular dystrophydesigning randomized trials69 %) relative(> 18 monthsbody mass indexnsaa total scoreambulatory motor functionquartile range 1dmd aged 4div >< pdaily corticosteroid regimens5 nsaa units6 years ).index yeardmd ).initially ambulatory6 years5 years16 yearsuz leuvensteroid typerun velocityreceiving prednisonereceiving corticosteroidsprediction errorsnovel therapiesmodifying therapiesmeter walkmean agelongitudinal datagenotype classfloor velocitydrug developmentdmd </discuss applicationsdata sourcecureduchenne ),controlled studiescontextualizing outcomesamong nambulatory patients31 %).<p>Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.</p>2025-06-27T17:40:29ZImageFigureinfo:eu-repo/semantics/publishedVersionimage10.1371/journal.pone.0325736.s009https://figshare.com/articles/figure/Predicted_Change_in_Imputed_and_Unimputed_NSAA_Total_Score_Up_to_60_Months_Post-Baseline_Baseline_among_Patients_with_Baseline_Age_10_Years_/29427473CC BY 4.0info:eu-repo/semantics/openAccessoai:figshare.com:article/294274732025-06-27T17:40:29Z
spellingShingle Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.
Francesco Muntoni (228210)
Medicine
Genetics
Environmental Sciences not elsewhere classified
Biological Sciences not elsewhere classified
Mathematical Sciences not elsewhere classified
Chemical Sciences not elsewhere classified
duchenne muscular dystrophy
designing randomized trials
69 %) relative
(> 18 months
body mass index
nsaa total score
ambulatory motor function
quartile range 1
dmd aged 4
div >< p
daily corticosteroid regimens
5 nsaa units
6 years ).
index year
dmd ).
initially ambulatory
6 years
5 years
16 years
uz leuven
steroid type
run velocity
receiving prednisone
receiving corticosteroids
prediction errors
novel therapies
modifying therapies
meter walk
mean age
longitudinal data
genotype class
floor velocity
drug development
dmd </
discuss applications
data source
cureduchenne ),
controlled studies
contextualizing outcomes
among n
ambulatory patients
31 %).
status_str publishedVersion
title Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.
title_full Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.
title_fullStr Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.
title_full_unstemmed Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.
title_short Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.
title_sort Predicted Change in Imputed and Unimputed NSAA Total Score Up to 60 Months Post-Baseline Baseline among Patients with Baseline Age < 10 Years.
topic Medicine
Genetics
Environmental Sciences not elsewhere classified
Biological Sciences not elsewhere classified
Mathematical Sciences not elsewhere classified
Chemical Sciences not elsewhere classified
duchenne muscular dystrophy
designing randomized trials
69 %) relative
(> 18 months
body mass index
nsaa total score
ambulatory motor function
quartile range 1
dmd aged 4
div >< p
daily corticosteroid regimens
5 nsaa units
6 years ).
index year
dmd ).
initially ambulatory
6 years
5 years
16 years
uz leuven
steroid type
run velocity
receiving prednisone
receiving corticosteroids
prediction errors
novel therapies
modifying therapies
meter walk
mean age
longitudinal data
genotype class
floor velocity
drug development
dmd </
discuss applications
data source
cureduchenne ),
controlled studies
contextualizing outcomes
among n
ambulatory patients
31 %).