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PubPharm (226)
1
Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients : An international study
enthalten in:
European journal of neurology
| 2024
von
Coratti, G.
|
Bovis, F.
|
Pera, M.
| +44
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2
Duchenne muscular dystrophy patients lacking the dystrophin isoforms Dp140 and Dp71 and mouse models lacking Dp140 have a more severe motor phenotype
enthalten in:
bioRxiv.org
| 2024
von
Chesshyre, M.
|
Ridout, D.
|
Hashimoto, Y.
| +12
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3
2-year Change in Revised Hammersmith Scale scores in a large cohort of untreated paediatric type 2 and 3 SMA participants
enthalten in:
bioRxiv.org
| 2024
von
Stimpson, G.
|
Ramsey, D.
|
Wolfe, A.
| +29
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4
Bulbar function in spinal muscular atrophy (SMA) : State of art and new challenges. 21st July 2023, Rome, Italy
enthalten in:
Neuromuscular disorders : NMD
| 2024
von
McGrattan, K.
|
Cerchiari, A.
|
Conway, E.
| +41
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5
Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3
enthalten in:
Journal of neuromuscular diseases
| 2024
von
Wolfe, A.
|
Stimpson, G.
|
Ramsey, D.
| +32
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6
Assessment of the upper limb function, strength, and mobility in treatment-naive children with spinal muscular atrophy Types 2 and 3
enthalten in:
Muscle & nerve
| 2024
von
Milev, E.
|
Selby, V.
|
Wolfe, A.
| +8
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7
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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8
Quantifying Variability in Motor Function in Duchenne Muscular Dystrophy : UK Centiles for the NorthStar Ambulatory Assessment, 10 m Walk Run Velocity and Rise from Floor Velocity in GC Treated Boys
enthalten in:
Journal of neuromuscular diseases
| 2024
von
Stimpson, G.
|
Ridout, D.
|
Wolfe, A.
| +8
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9
The emerging spectrum of neurodevelopmental comorbidities in early-onset Spinal Muscular Atrophy
enthalten in:
European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
| 2024
von
Baranello, G.
|
Neurodevelopment in SMA Working Group
|
Roy, S.
| +55
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10
Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1 : real-world evidence from 6 infusion centres in the United Kingdom
enthalten in:
The Lancet regional health. Europe
| 2024
von
Gowda, V.
|
Atherton, M.
|
Murugan, A.
| +34
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