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PubPharm (7)
1
Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping : A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial
enthalten in:
Nucleic acid therapeutics
| 2022
von
Servais, L.
|
Mercuri, E.
|
Straub, V.
| +29
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2
Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping : A randomized, double-blind, placebo-controlled, dose-titration trial
enthalten in:
Muscle & nerve
| 2021
von
Wagner, K.
|
Kuntz, N.
|
Koenig, E.
| +4
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3
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping : PROMOVI Trial
enthalten in:
Journal of neuromuscular diseases
| 2021
von
McDonald, C.
|
Shieh, P.
|
Abdel-Hamid, H.
| +12
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4
The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
enthalten in:
Acta neuropathologica communications
| 2021
von
Scaglioni, D.
|
Catapano, F.
|
Ellis, M.
| +17
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5
The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
enthalten in:
Acta Neuropathologica Communications
| 2021
von
Scaglioni, D.
|
Catapano, F.
|
Ellis, M.
| +17
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6
Safety, tolerability, and pharmacokinetics of casimersen in patients with D uchenne muscular dystrophy amenable to exon45 skipping : A randomized, double‐blind, placebo‐controlled, dose‐titration trial
enthalten in:
Muscle & Nerve
| 2021
von
Wagner, K.
|
Kuntz, N.
|
Koenig, E.
| +4
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7
Randomized phase I clinical trial of anti-α-synuclein antibody BIIB054
enthalten in:
Movement disorders : official journal of the Movement Disorder Society
| 2019
von
Brys, M.
|
Fanning, L.
|
Hung, S.
| +15
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1
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Medienart
7
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7
E-Artikel
7
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Zeitschriftentitel
1
Acta Neuropathologica Communications
1
Acta neuropathologica communications
1
Journal of neuromuscular diseases
1
Movement disorders : official journal of the Mo...
1
Muscle & Nerve
1
Muscle & nerve
1
Nucleic acid therapeutics
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Thema
5
Dystrophin
5
Journal Article
4
Research Support, Non-U.S. Gov't
3
Clinical Trial, Phase I
3
Multicenter Study
3
Muscular dystrophy
3
Oligonucleotides
3
Randomized Controlled Trial
2
033072U4MZ
2
Clinical Trial, Phase II
2
Clinical trial
2
Duchenne muscular dystrophy
2
Genetic therapies
2
Golodirsen
2
Immunofluorescence
2
exon skipping
2
golodirsen
1
146888-27-9
1
AIW6036FAS
1
Antibodies, Monoclonal
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Erscheinungszeitraum
6
2020-
1
2010-2019
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6
Englisch
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