Page 36 - Read Online
P. 36
Page 351 Gonzalez Castillo et al. J Transl Genet Genom. 2025;9:338-51 https://dx.doi.org/10.20517/jtgg.2025.57
2019. JAMA Netw Open. 2022;5:e226479. DOI PubMed PMC
72. Mazzone E, Martinelli D, Berardinelli A, et al. North Star Ambulatory assessment, 6-minute walk test and timed items in ambulant
boys with Duchenne muscular dystrophy. Neuromuscul Disord. 2010;20:712-6. DOI
73. Mayhew AG, Cano SJ, Scott E, et al. Detecting meaningful change using the North Star Ambulatory assessment in Duchenne
muscular dystrophy. Dev Med Child Neurol. 2013;55:1046-52. DOI
74. McDonald CM, Henricson EK, Abresch RT, et al. The 6-minute walk test and other clinical endpoints in duchenne muscular
dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study. Muscle Nerve.
2013;48:357-68. DOI PubMed PMC
75. Henricson E, Abresch R, Han JJ, et al. The 6-minute walk test and person-reported outcomes in boys with Duchenne muscular
dystrophy and typically developing controls: longitudinal comparisons and clinically-meaningful changes over one year. PLoS Curr.
2013;5:ecurrents. DOI PubMed PMC
76. Ayyar Gupta V, Pitchforth JM, Domingos J, et al. Determining minimal clinically important differences in the North Star Ambulatory
Assessment (NSAA) for patients with Duchenne muscular dystrophy. PLoS One. 2023;18:e0283669. DOI PubMed PMC
77. Muntoni F, Signorovitch J, Sajeev G, et al. Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): a multi-
center study. PLoS One. 2024;19:e0304984. DOI PubMed PMC
78. Henricson E, Abresch R, Han JJ, et al. Percent-predicted 6-minute walk distance in duchenne muscular dystrophy to account for
maturational influences. PLoS Curr. 2012;4:RRN1297. DOI
79. Mayhew A, Mazzone ES, Eagle M, et al. Development of the performance of the upper limb module for duchenne muscular
dystrophy. Dev Med Child Neurol. 2013;55:1038-45. DOI
80. Mayhew AG, Coratti G, Mazzone ES, et al. Performance of upper limb module for duchenne muscular dystrophy. Dev Med Child
Neurol. 2020;62:633-9. DOI
81. Bushby K, Finkel R, Wong B, et al. Safety and effectiveness of ataluren in patients with nonsense mutation Duchenne muscular
dystrophy: 2022 interim analysis from the STRIDE Registry compared with the CINRG Duchenne Natural History Study. J Neurol.
2023;270:2885-95. DOI
82. Henricson EK, Abresch RT, Cnaan A, et al. The cooperative international neuromuscular research group Duchenne natural history
study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as
measured by manual muscle testing and other commonly used clinical trial outcome measures. Muscle Nerve. 2013;48:55-67. DOI
83. Horrow C, Kesselheim AS. Confronting high costs and clinical uncertainty: innovative payment models for gene therapies. Health Aff.
2023;42:1532-40. DOI PubMed

