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PubPharm (122)
1
Regional and bilateral MRI and gene signatures in facioscapulohumeral dystrophy : implications for clinical trial design and mechanisms of disease progression
enthalten in:
Human molecular genetics
| 2024
von
Wong, C.
|
Friedman, S.
|
Snider, L.
| +13
UpdateOf: bioRxiv. 2023 Feb 20;:. - PMID 36865168
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2
Correction to : A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot-Marie-Tooth type 1A
enthalten in:
Orphanet journal of rare diseases
| 2024
von
Attarian, S.
|
Young, P.
|
Brannagan, T.
| +17
ErratumFor: Orphanet J Rare Dis. 2021 Oct 16;16(1):433. - PMID 34656144
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3
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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4
Correction to: A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot–Marie–Tooth type 1A
enthalten in:
Orphanet journal of rare diseases
| 2024
von
Attarian, S.
|
Young, P.
|
Brannagan, T.
| +17
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5
Longitudinal course of neurofilament light chain levels in amyotrophic lateral sclerosis-insights from a completed randomized controlled trial with rasagiline
enthalten in:
European journal of neurology
| 2024
von
Witzel, S.
|
Statland, J.
|
Steinacker, P.
| +5
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6
Longitudinal course of neurofilament light chain levels in amyotrophic lateral sclerosis—insights from a completed randomized controlled trial with rasagiline
enthalten in:
European Journal of Neurology
| 2024
von
Witzel, S.
|
Statland, J.
|
Steinacker, P.
| +5
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7
Phase 2 trial in acetylcholine receptor antibody-positive myasthenia gravis of transition from intravenous to subcutaneous immunoglobulin : The MGSCIg study
enthalten in:
European journal of neurology
| 2023
von
Pasnoor, M.
|
Bril, V.
|
Levine, T.
| +12
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8
Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1 : a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
enthalten in:
The Lancet. Neurology
| 2023
von
Thornton, C.
|
Moxley, R.
|
Eichinger, K.
| +24
CommentIn: Lancet Neurol. 2023 Mar;22(3):191-192. - PMID 36804078
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9
Validation of the association between MRI and gene signatures in facioscapulohumeral dystrophy muscle: implications for clinical trial design
enthalten in:
bioRxiv.org
| 2023
von
Wong, C.
|
Friedman, S.
|
Snider, L.
| +13
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10
Validation of the association between MRI and gene signatures in facioscapulohumeral dystrophy muscle : implications for clinical trial design
enthalten in:
bioRxiv : the preprint server for biology
| 2023
von
Wong, C.
|
Friedman, S.
|
Snider, L.
| +13
UpdateIn: Hum Mol Genet. 2024 Jan 24;:. - PMID 38268317
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122
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Muscle & nerve
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8
Orphanet journal of rare diseases
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70
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38
Research Support, N.I.H., Extramural
34
Research Support, Non-U.S. Gov't
13
Multicenter Study
12
Randomized Controlled Trial
12
Review
12
facioscapulohumeral muscular dystrophy
10
Biomarkers
7
Motor neuron disease
7
Muscular dystrophy
6
Amyotrophic lateral sclerosis
6
Mexiletine
6
muscular dystrophy
5
Homeodomain Proteins
5
Observational Study
5
outcome measures
4
1U511HHV4Z
4
Chloride Channels
4
Clinical trial
4
Comparative Study
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Erscheinungszeitraum
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2020-
61
2010-2019
8
2000-2009
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