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PubPharm (289)
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
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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3
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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4
Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy : Findings from the RESTORE Registry
enthalten in:
Journal of neuromuscular diseases
| 2024
von
Servais, L.
|
Day, J.
|
De Vivo, D.
| +16
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5
Combination disease-modifying treatment in spinal muscular atrophy : A proposed classification
enthalten in:
Annals of clinical and translational neurology
| 2023
von
Proud, C.
|
Mercuri, E.
|
Finkel, R.
| +11
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6
Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy : 5-year update of the NURTURE study
enthalten in:
Muscle & nerve
| 2023
von
Crawford, T.
|
Swoboda, K.
|
De Vivo, D.
| +23
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7
Nusinersen Treatment of Children with Later-Onset Spinal Muscular Atrophy and Scoliosis Is Associated with Improvements or Stabilization of Motor Function
enthalten in:
Journal of clinical medicine
| 2023
von
Dunaway Young, S.
|
Montes, J.
|
Glanzman, A.
| +11
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8
Perceived exertion is not a substitute for fatiguability in spinal muscular atrophy
enthalten in:
Muscle & nerve
| 2023
von
Cheng, R.
|
Feng, J.
|
Goodwin, A.
| +6
CommentIn: Muscle Nerve. 2023 Jul;68(1):6-7. - PMID 37086214
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9
Systemic Reduction of Glut1 Normalizes Retinal Dysfunction, Inflammation, and Oxidative Stress in the Retina of Spontaneous Type 2 Diabetic Mice
enthalten in:
The American journal of pathology
| 2023
von
Aiello, J.
|
Bogart, M.
|
Chan, W.
| +6
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10
Measuring Fatigue and Fatigability in Spinal Muscular Atrophy (SMA) : Challenges and Opportunities
enthalten in:
Journal of clinical medicine
| 2023
von
Rodriguez-Torres, R.
|
Uher, D.
|
Gay, E.
| +8
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Muscle & nerve
25
Journal of child neurology
16
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12
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Neurology
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Neuromuscular disorders : NMD
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Annals of Clinical and Translational Neurology
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Molecular genetics and metabolism
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3
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3
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Thema
167
Journal Article
108
Research Support, Non-U.S. Gov't
62
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39
Glucose Transporter Type 1
28
Case Reports
25
spinal muscular atrophy
21
SLC2A1 protein, human
21
Spinal muscular atrophy
20
Monosaccharide Transport Proteins
19
Multicenter Study
17
Glucose
17
IY9XDZ35W2
17
Research Support, U.S. Gov't, P.H.S.
16
DNA, Mitochondrial
16
Oligonucleotides
15
nusinersen
14
5Z9SP3X666
13
Review
10
outcome measure
8
Research Support, U.S. Gov't, Non-P.H.S.
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Erscheinungszeitraum
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2020-
132
2010-2019
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