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PubPharm (105)
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
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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5
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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6
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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7
Engineering Skeletal Muscle Grafts with PAX7::GFP-Sorted Human Pluripotent Stem Cell-Derived Myogenic Progenitors on Fibrin Microfiber Bundles for Tissue Regeneration
enthalten in:
Bioengineering (Basel, Switzerland)
| 2022
von
Somers, S.
|
Gilbert-Honick, J.
|
Choi, I.
| +8
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8
Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy
enthalten in:
Scientific reports
| 2022
von
Sherlock, S.
|
Palmer, J.
|
Wagner, K.
| +8
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9
Quantitative Muscle Analysis in FSHD Using Whole-Body Fat-Referenced MRI : Composite Scores for Longitudinal and Cross-sectional Analysis
enthalten in:
Neurology
| 2022
von
Mellion, M.
|
Widholm, P.
|
Karlsson, M.
| +16
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10
Quantitative muscle analysis in facioscapulohumeral muscular dystrophy using whole-body fat-referenced MRI : Protocol development, multicenter feasibility, and repeatability
enthalten in:
Muscle & nerve
| 2022
von
Widholm, P.
|
Ahlgren, A.
|
Karlsson, M.
| +13
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Thema: Journal Article
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Thema
Journal Article
61
Research Support, Non-U.S. Gov't
39
Research Support, N.I.H., Extramural
16
Myostatin
16
Review
11
Randomized Controlled Trial
10
Duchenne muscular dystrophy
9
Biomarkers
9
Clinical Trial, Phase II
9
Dystrophin
8
muscular dystrophy
7
Multicenter Study
7
Transforming Growth Factor beta
6
Activin Receptors, Type II
6
EC 2.7.11.30
6
Homeodomain Proteins
6
Mstn protein, mouse
6
Research Support, U.S. Gov't, P.H.S.
6
facioscapulohumeral muscular dystrophy
5
Case Reports
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Erscheinungszeitraum
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2020-
53
2010-2019
13
2000-2009
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105
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