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PubPharm (21)
1
Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy
enthalten in:
Journal of neuromuscular diseases
| 2024
von
Zaidman, C.
|
Goedeker, N.
|
Aqul, A.
| +11
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2
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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3
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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4
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy : 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR)
enthalten in:
Annals of neurology
| 2023
von
Zaidman, C.
|
Proud, C.
|
McDonald, C.
| +15
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5
Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources
enthalten in:
Advances in therapy
| 2023
von
Youn, B.
|
Proud, C.
|
Wang, N.
| +6
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6
Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources
enthalten in:
Advances in therapy
| 2023
von
Youn, B.
|
Proud, C.
|
Wang, N.
| +6
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7
Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources
enthalten in:
Advances in therapy
| 2023
von
Youn, B.
|
Proud, C.
|
Wang, N.
| +6
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8
Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources
enthalten in:
Advances in therapy
| 2023
von
Youn, B.
|
Proud, C.
|
Wang, N.
| +6
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9
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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10
Onasemnogene abeparvovec for the treatment of spinal muscular atrophy
enthalten in:
Expert opinion on biological therapy
| 2022
von
McMillan, H.
|
Proud, C.
|
Farrar, M.
| +3
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Journal of neuromuscular diseases
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11
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Electronic health records
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Nusinersen
4
Real-world adherence
4
Research Support, Non-U.S. Gov't
4
Spinal muscular atrophy
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spinal muscular atrophy
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gene therapy
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onasemnogene abeparvovec
2
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newborn screening
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nusinersen
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AAV9 vector-based gene replacement therapy
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Erscheinungszeitraum
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2020-
6
2010-2019
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