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PubPharm (268)
1
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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2
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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3
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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4
Can the CHOP-INTEND be used as An Outcome Measure in the First Months of Age? Implications for Clinical Trials and Real World Data
enthalten in:
Journal of neuromuscular diseases
| 2024
von
Cutrona, C.
|
de Sanctis, R.
|
Coratti, G.
| +14
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5
The Clinical Development of Taldefgrobep Alfa : An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy
enthalten in:
Neurology and therapy
| 2024
von
Muntoni, F.
|
Byrne, B.
|
McMillan, H.
| +114
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6
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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7
Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease
enthalten in:
Journal of neuromuscular diseases
| 2024
von
McDonald, C.
|
Camino, E.
|
Escandon, R.
| +8
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8
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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9
Genetic analysis and natural history of Charcot-Marie-Tooth disease CMTX1 due to GJB1 variants
enthalten in:
Brain : a journal of neurology
| 2023
von
Record, C.
|
Skorupinska, M.
|
Laura, M.
| +71
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10
Validation of the parent-proxy version of the pediatric Charcot-Marie-Tooth disease quality of life instrument for children aged 0-7 years
enthalten in:
Journal of the peripheral nervous system : JPNS
| 2023
von
Wu, T.
|
Finkel, R.
|
Siskind, C.
| +9
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nusinersen
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spinal muscular atrophy
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Dystrophin
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Survival of Motor Neuron 2 Protein
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Glucocorticoids
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