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PubPharm (117)
1
Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids
enthalten in:
European journal of neurology
| 2024
von
Schiava, M.
|
Lofra, R.
|
Bourke, J.
| +20
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2
266th ENMC International Workshop : Remote delivery of clinical care and validation of remote clinical outcome assessments in neuromuscular disorders: A response to COVID-19 and proactive planning for the future. Hoofddorp, The Netherlands, 1-3 April 2022
enthalten in:
Neuromuscular disorders : NMD
| 2023
von
Alfano, L.
|
James, M.
|
Ramdharry, G.
| +25
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3
Validation of the North Star Assessment for Limb-Girdle Type Muscular Dystrophies
enthalten in:
Physical therapy
| 2022
von
James, M.
|
Alfano, L.
|
Muni-Lofra, R.
| +12
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4
Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy
enthalten in:
Neuromuscular disorders : NMD
| 2022
von
Mayhew, A.
|
Moat, D.
|
McDermott, M.
| +49
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5
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy : A Randomized Clinical Trial
enthalten in:
JAMA
| 2022
von
Guglieri, M.
|
Bushby, K.
|
McDermott, M.
| +61
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6
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2) : a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial
enthalten in:
Lancet (London, England)
| 2022
von
McDonald, C.
|
Marbán, E.
|
Hendrix, S.
| +54
CommentIn: Lancet. 2022 Mar 12;399(10329):1024-1025. - PMID 35279246
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7
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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8
Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale
enthalten in:
Annals of neurology
| 2021
von
Jacobs, M.
|
James, M.
|
Lowes, L.
| +40
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9
A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy : Results of the PolarisDMD Trial
enthalten in:
Journal of neuromuscular diseases
| 2021
von
Finkel, R.
|
McDonald, C.
|
Lee Sweeney, H.
| +24
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10
Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease : A Response to the COVID-19 Global Pandemic
enthalten in:
Frontiers in genetics
| 2021
von
James, M.
|
Rose, K.
|
Alfano, L.
| +3
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Neuromuscular disorders : NMD
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Duchenne muscular dystrophy
5
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Codon, Nonsense
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K16AME9I3V
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4
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ataluren
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muscular dystrophy
3
Adrenal Cortex Hormones
3
Clinical Trial, Phase III
3
Congenital Ptosis
3
Copy Number Abnormality
3
Dystrophin
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