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PubPharm (29)
1
Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy
enthalten in:
Scientific reports
| 2022
von
Sherlock, S.
|
Palmer, J.
|
Wagner, K.
| +8
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2
Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy : a phase 2 trial of domagrozumab
enthalten in:
Journal of neurology
| 2022
von
Sherlock, S.
|
Palmer, J.
|
Wagner, K.
| +13
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3
Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab
enthalten in:
Journal of neurology
| 2022
von
Sherlock, S.
|
Palmer, J.
|
Wagner, K.
| +13
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4
Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab
enthalten in:
Journal of neurology
| 2022
von
Sherlock, S.
|
Palmer, J.
|
Wagner, K.
| +13
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5
Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab
enthalten in:
Journal of neurology
| 2022
von
Sherlock, S.
|
Palmer, J.
|
Wagner, K.
| +13
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6
A homozygous CAP2 pathogenic variant in a neonate presenting with rapidly progressive cardiomyopathy and nemaline rods
enthalten in:
American journal of medical genetics. Part A
| 2022
von
Gurunathan, S.
|
Sebastian, J.
|
Baker, J.
| +7
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7
A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment
enthalten in:
Journal of neuromuscular diseases
| 2022
von
Mitelman, O.
|
Abdel-Hamid, H.
|
Byrne, B.
| +15
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8
A homozygous CAP2 pathogenic variant in a neonate presenting with rapidly progressive cardiomyopathy and nemaline rods
enthalten in:
American Journal of Medical Genetics Part C: Seminars in Medical Genetics
| 2022
von
Gurunathan, S.
|
Sebastian, J.
|
Baker, J.
| +7
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9
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy
enthalten in:
Journal of comparative effectiveness research
| 2021
von
Shieh, P.
|
Elfring, G.
|
Trifillis, P.
| +111
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10
Corrigendum to "Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy" [Neuromuscular Disorders, Vol. 30 (6) 2020, 492-502
enthalten in:
Neuromuscular disorders : NMD
| 2021
von
Wagner, K.
|
Abdel-Hamid, H.
|
Mah, J.
| +21
ErratumFor: Neuromuscul Disord. 2020 Jun;30(6):492-502. - PMID 32522498
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Muscle & nerve
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Journal of neurology
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American journal of medical genetics. Part A
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Neuromuscular disorders : NMD
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Spine deformity
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Journal Article
13
Duchenne muscular dystrophy
7
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7
Research Support, Non-U.S. Gov't
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Biomarkers
5
Randomized Controlled Trial
5
newborn screening
4
Domagrozumab
4
Imaging
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MRI
4
Multicenter Study
4
Muscular dystrophy
4
Neuromuscular disease
3
AIW6036FAS
3
Becker muscular dystrophy
3
Dystrophin
3
Morpholinos
3
Research Support, N.I.H., Extramural
3
eteplirsen
3
parental attitudes
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Erscheinungszeitraum
14
2020-
15
2010-2019
Erscheinungsjahr(e)
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26
Englisch
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