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PubPharm (280)
1
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS) : a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
enthalten in:
The Lancet. Neurology
| 2024
von
Mercuri, E.
|
Vilchez, J.
|
Boespflug-Tanguy, O.
| +25
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2
Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls
enthalten in:
Muscle & nerve
| 2024
von
Iff, J.
|
Done, N.
|
Tuttle, E.
| +6
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3
Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy
enthalten in:
Pediatric neurology
| 2024
von
Mendell, J.
|
Proud, C.
|
Zaidman, C.
| +8
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4
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy : A Randomized Controlled Trial
enthalten in:
Neurology
| 2024
von
Dang, U.
|
Damsker, J.
|
Guglieri, M.
| +41
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5
Gait Event Detection and Travel Distance Using Waist-Worn Accelerometers across a Range of Speeds : Automated Approach
enthalten in:
Sensors (Basel, Switzerland)
| 2024
von
Ramli, A.
|
Liu, X.
|
Berndt, K.
| +11
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6
Gait Characterization in Duchenne Muscular Dystrophy (DMD) Using a Single-Sensor Accelerometer : Classical Machine Learning and Deep Learning Approaches
enthalten in:
Sensors (Basel, Switzerland)
| 2024
von
Ramli, A.
|
Liu, X.
|
Berndt, K.
| +13
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7
Findings from the Longitudinal CINRG Becker Natural History Study
enthalten in:
Journal of neuromuscular diseases
| 2024
von
Clemens, P.
|
Gordish-Dressman, H.
|
Niizawa, G.
| +17
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8
The IAAM LTBP4 Haplotype is Protective Against Dystrophin-Deficient Cardiomyopathy
enthalten in:
Journal of neuromuscular diseases
| 2024
von
Bello, L.
|
Sabbatini, D.
|
Fusto, A.
| +36
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9
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy : 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR)
enthalten in:
Annals of neurology
| 2023
von
Zaidman, C.
|
Proud, C.
|
McDonald, C.
| +15
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10
Sleep Disordered Breathing in Children with Neuromuscular Disease
enthalten in:
Children (Basel, Switzerland)
| 2023
von
Chidambaram, A.
|
Jhawar, S.
|
McDonald, C.
| +1
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Muscle & nerve
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66
Duchenne muscular dystrophy
65
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57
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30
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13
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11
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9
K16AME9I3V
9
ataluren
8
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8
Codon, Nonsense
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