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PubPharm (146)
1
Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4) : a randomised, double-blind, placebo-controlled phase 2b trial
enthalten in:
The Lancet. Neurology
| 2024
von
Tawil, R.
|
Wagner, K.
|
Hamel, J.
| +36
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2
Racial and ethnic diversity in global neuroscience clinical trials
enthalten in:
Contemporary clinical trials communications
| 2024
von
Rutten-Jacobs, L.
|
McIver, T.
|
Reyes, A.
| +5
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3
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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4
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.
| +13
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5
Correction to : Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study
enthalten in:
Neurology and therapy
| 2023
von
Chiriboga, C.
|
Bruno, C.
|
Duong, T.
| +114
ErratumFor: Neurol Ther. 2023 Apr;12(2):543-557. - PMID 36780114
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6
Lean tissue mass measurements by dual-energy X-ray absorptiometry and associations with strength and functional outcome measures in facioscapulohumeral muscular dystrophy
enthalten in:
Neuromuscular disorders : NMD
| 2023
von
Wang, L.
|
Leung, D.
|
Wagner, K.
| +20
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7
Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study
enthalten in:
Neurology and Therapy
| 2023
von
Chiriboga, C.
|
Bruno, C.
|
Duong, T.
| +13
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8
Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study
enthalten in:
Neurology and Therapy
| 2023
von
Chiriboga, C.
|
Bruno, C.
|
Duong, T.
| +13
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9
Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials
enthalten in:
Human gene therapy
| 2023
von
Chamberlain, J.
|
Robb, M.
|
Braun, S.
| +11
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10
Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy : An Interim Analysis from the JEWELFISH Study
enthalten in:
Neurology and therapy
| 2023
von
Chiriboga, C.
|
Bruno, C.
|
Duong, T.
| +114
ErratumIn: Neurol Ther. 2023 Oct;12(5):1799-1801. - PMID 37395990
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Medienart: E-Ressourcen
Medienart
146
Aufsätze
146
E-Artikel
E-Ressourcen
Zeitschriftentitel
12
Muscle & nerve
7
Neuromuscular disorders : NMD
6
Muscle & Nerve
5
Neurology and Therapy
5
bioRxiv.org
4
Annals of neurology
4
Europäisches Patentamt
4
Skeletal muscle
3
Annals of clinical and translational neurology
3
BMC neurology
3
Human molecular genetics
3
Journal of neurology
3
Journal of neuromuscular diseases
3
Molecular therapy : the journal of the American...
3
Neurology
3
Neurology and therapy
3
Pediatrics
3
The Journal of biological chemistry
2
Annals of Clinical and Translational Neurology
2
Clinical epigenetics
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Thema
97
Journal Article
60
Research Support, Non-U.S. Gov't
39
Research Support, N.I.H., Extramural
14
Biomarkers
14
Review
13
Duchenne muscular dystrophy
13
Myostatin
11
Randomized Controlled Trial
9
Clinical Trial, Phase II
9
Dystrophin
8
Muscular dystrophy
8
muscular dystrophy
7
Homeodomain Proteins
7
Multicenter Study
6
Activin Receptors, Type II
6
EC 2.7.11.30
6
facioscapulohumeral muscular dystrophy
5
Case Reports
5
Comment
5
MRI
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Erscheinungszeitraum
70
2020-
64
2010-2019
12
2000-2009
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Sprache
135
Englisch
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