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PubPharm (188)
1
Experimental Neurotherapeutics : Surfing the Tidal Wave of New Opportunities
enthalten in:
Annals of neurology
| 2023
von
Reoma, L.
|
Busza, A.
|
Mouradian, M.
| +9
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2
Experimental Neurotherapeutics : Surfing the Tidal Wave of New Opportunities
enthalten in:
Annals of Neurology
| 2023
von
Reoma, L.
|
Busza, A.
|
Mouradian, M.
| +9
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3
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy
enthalten in:
Journal of comparative effectiveness research
| 2021
von
Shieh, P.
|
Elfring, G.
|
Trifillis, P.
| +111
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4
Clinical Design and Analysis Strategies for the Development of Gene Therapies : Considerations for Quantitative Drug Development in the Age of Genetic Medicine
enthalten in:
Clinical pharmacology and therapeutics
| 2021
von
McIntosh, A.
|
Sverdlov, O.
|
Yu, L.
| +1
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5
Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue
enthalten in:
Nature medicine
| 2021
von
Thomsen, G.
|
Burghes, A.
|
Hsieh, C.
| +16
CommentIn: Front Cell Neurosci. 2022 May 17;16:916065. - PMID 35656408
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6
Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy
enthalten in:
Journal of hepatology
| 2021
von
Chand, D.
|
Mohr, F.
|
McMillan, H.
| +7
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7
Clinical Design and Analysis Strategies for the Development of Gene Therapies : Considerations for Quantitative Drug Development in the Age of Genetic Medicine
enthalten in:
Clinical Pharmacology & Therapeutics
| 2021
von
McIntosh, A.
|
Sverdlov, O.
|
Yu, L.
| +1
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8
A call for global action for rare diseases in Africa
enthalten in:
Nature genetics
| 2020
von
Baynam, G.
|
Groft, S.
|
van der Westhuizen, F.
| +47
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9
Resources to assist in the transition to a single IRB model for multisite clinical trials
enthalten in:
Contemporary clinical trials communications
| 2019
von
Hahn, C.
|
Kaufmann, P.
|
Bang, S.
| +1
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10
Model consent clauses for rare disease research
enthalten in:
BMC medical ethics
| 2019
von
Nguyen, M.
|
Goldblatt, J.
|
Isasi, R.
| +22
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Archives of neurology
14
Journal of child neurology
14
Muscle & nerve
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BMC medical ethics
3
Journal of the neurological sciences
3
Neurotherapeutics : the journal of the American...
3
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38
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23
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11
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10
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8
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8
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8
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6
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spinal muscular atrophy
5
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4
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