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PubPharm (216)
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
SNUPN deficiency causes a recessive muscular dystrophy due to RNA mis-splicing and ECM dysregulation
enthalten in:
Nature communications
| 2024
von
Nashabat, M.
|
Nabavizadeh, N.
|
Saraçoğlu, H.
| +56
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3
Parental Experiences with Newborn Screening and Gene Replacement Therapy for Spinal Muscular Atrophy
enthalten in:
Journal of neuromuscular diseases
| 2024
von
Meyer, A.
|
Connolly, A.
|
Vannatta, K.
| +6
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4
Findings from the Longitudinal CINRG Becker Natural History Study
enthalten in:
Journal of neuromuscular diseases
| 2024
von
Clemens, P.
|
Gordish-Dressman, H.
|
Niizawa, G.
| +17
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5
Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in Duchenne Muscular Dystrophy (DMD) : Meeting Report from the 3rd International Muscle-Bone Interactions Meeting 7th and 14th November 2022
enthalten in:
Journal of neuromuscular diseases
| 2024
von
Phung, K.
|
Crabtree, N.
|
Connolly, A.
| +17
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6
Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio
enthalten in:
Neuromuscular disorders : NMD
| 2024
von
Waldrop, M.
|
Chagat, S.
|
Storey, M.
| +19
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7
Pamrevlumab, a Fully Human Monoclonal Antibody Targeting Connective Tissue Growth Factor, for Non-Ambulatory Patients with Duchenne Muscular Dystrophy
enthalten in:
Journal of neuromuscular diseases
| 2023
von
Connolly, A.
|
Zaidman, C.
|
Brandsema, J.
| +6
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8
Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy : Results From the Phase 2, Open-Label, 4-Year Extension Study
enthalten in:
Journal of neuromuscular diseases
| 2023
von
Clemens, P.
|
Rao, V.
|
Connolly, A.
| +8
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9
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy : Phase I Ascending-Dose Study (STRONG)
enthalten in:
Journal of neuromuscular diseases
| 2023
von
Finkel, R.
|
Darras, B.
|
Mendell, J.
| +14
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10
Testing preexisting antibodies prior to AAV gene transfer therapy : rationale, lessons and future considerations
enthalten in:
Molecular therapy. Methods & clinical development
| 2022
von
Mendell, J.
|
Connolly, A.
|
Lehman, K.
| +5
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