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PubPharm (91)
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
Communicative development inventory in type 1 and presymptomatic infants with spinal muscular atrophy : a cohort study
enthalten in:
Archives of disease in childhood
| 2024
von
Buchignani, B.
|
Cicala, G.
|
Cumbo, F.
| +21
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3
Early neurological signs in infants identified through neonatal screening for SMA : do they predict outcome?
enthalten in:
European journal of pediatrics
| 2024
von
Pane, M.
|
Stanca, G.
|
Ticci, C.
| +25
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4
Early treatment of type II SMA slows rate of progression of scoliosis
enthalten in:
Journal of neurology, neurosurgery, and psychiatry
| 2024
von
Coratti, G.
|
Lenkowicz, J.
|
Pera, M.
| +23
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5
Clinical Phenotype of Pediatric and Adult Patients With Spinal Muscular Atrophy With Four SMN2 Copies : Are They Really All Stable?
enthalten in:
Annals of neurology
| 2023
von
Ricci, M.
|
Cicala, G.
|
Capasso, A.
| +72
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6
Correction to : Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study
enthalten in:
Neurology and therapy
| 2023
von
Chiriboga, C.
|
Bruno, C.
|
Duong, T.
| +114
ErratumFor: Neurol Ther. 2023 Apr;12(2):543-557. - PMID 36780114
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7
New therapies for spinal muscular atrophy : where we stand and what is next
enthalten in:
European journal of pediatrics
| 2023
von
Antonaci, L.
|
Pera, M.
|
Mercuri, E.
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8
Risdiplam in types 2 and 3 spinal muscular atrophy : A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment
enthalten in:
European journal of neurology
| 2023
von
Mercuri, E.
|
Baranello, G.
|
Boespflug-Tanguy, O.
| +19
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9
Caregivers' Expectations on Possible Functional Changes following Disease-Modifying Treatment in Type II and III Spinal Muscular Atrophy : A Comparative Study
enthalten in:
Journal of clinical medicine
| 2023
von
Pera, M.
|
Coratti, G.
|
Casiraghi, J.
| +9
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10
Type I spinal muscular atrophy patients treated with nusinersen : 4-year follow-up of motor, respiratory and bulbar function
enthalten in:
European journal of neurology
| 2023
von
Pane, M.
|
Coratti, G.
|
Sansone, V.
| +31
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12
European journal of pediatrics
11
Neuromuscular disorders : NMD
7
Orphanet journal of rare diseases
5
Muscle & nerve
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4
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Thema
58
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29
Research Support, Non-U.S. Gov't
27
Spinal muscular atrophy
10
Multicenter Study
9
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8
Neuromuscular disorders
8
Oligonucleotides
7
5Z9SP3X666
7
nusinersen
7
spinal muscular atrophy
6
Neonate
5
Risdiplam
4
76RS4S2ET1
4
Azo Compounds
4
Children
4
Fatty infiltration
4
Magnetic Resonance Imaging
4
Muscle atrophy
4
Natural history
4
Pyrimidines
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Erscheinungszeitraum
52
2020-
39
2010-2019
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