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PubPharm (835)
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
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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4
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
ErratumIn: Lancet Neurol. 2024 Apr 18;:. - PMID 38643778
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5
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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6
Hospital admissions from the emergency department of adult patients affected by myopathies
enthalten in:
European journal of neurology
| 2024
von
Monforte, M.
|
Torchia, E.
|
Bortolani, S.
| +12
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7
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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8
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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9
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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10
Ligamentous laxity in children with achondroplasia : Prevalence, joint involvement, and implications for early intervention strategies
enthalten in:
European journal of medical genetics
| 2024
von
Romeo, D.
|
Pironi, V.
|
Velli, C.
| +12
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Neuromuscular disorders : NMD
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14
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12
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11
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11
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11
The journal of pediatrics
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432
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Spinal muscular atrophy
61
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51
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43
Duchenne muscular dystrophy
38
Oligonucleotides
32
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31
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29
5Z9SP3X666
29
nusinersen
26
Randomized Controlled Trial
23
spinal muscular atrophy
19
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18
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13
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13
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13
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