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PubPharm (115)
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
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy : A Randomized Controlled Trial
enthalten in:
Neurology
| 2024
von
Dang, U.
|
Damsker, J.
|
Guglieri, M.
| +41
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3
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy : results from a substudy of the ASPIRO open-label clinical trial
enthalten in:
EBioMedicine
| 2024
von
Lawlor, M.
|
Schoser, B.
|
Margeta, M.
| +36
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4
Recurrent de novo SPTLC2 variant causes childhood-onset amyotrophic lateral sclerosis (ALS) by excess sphingolipid synthesis
enthalten in:
Journal of neurology, neurosurgery, and psychiatry
| 2024
von
Syeda, S.
|
Lone, M.
|
Mohassel, P.
| +23
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5
Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy : Findings from the RESTORE Registry
enthalten in:
Journal of neuromuscular diseases
| 2024
von
Servais, L.
|
Day, J.
|
De Vivo, D.
| +16
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6
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO) : a multinational, open-label, dose-escalation trial
enthalten in:
The Lancet. Neurology
| 2023
von
Shieh, P.
|
Kuntz, N.
|
Dowling, J.
| +30
CommentIn: Lancet Neurol. 2023 Dec;22(12):1089-1091. - PMID 37977700
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7
Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies
enthalten in:
bioRxiv.org
| 2023
von
Fischer, R.
|
Furlong, P.
|
Kennedy, A.
| +11
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8
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks : A Phase 3 Randomized Clinical Trial
enthalten in:
JAMA neurology
| 2023
von
Kishnani, P.
|
Diaz-Manera, J.
|
Toscano, A.
| +125
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9
Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status
enthalten in:
Journal of comparative effectiveness research
| 2023
von
McDonald, C.
|
Marden, J.
|
Shieh, P.
| +8
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10
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy : Phase I Ascending-Dose Study (STRONG)
enthalten in:
Journal of neuromuscular diseases
| 2023
von
Finkel, R.
|
Darras, B.
|
Mendell, J.
| +14
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Medienart: Aufsätze
Medienart
Aufsätze
93
E-Artikel
93
E-Ressourcen
22
Gedruckte Aufsätze
Zeitschriftentitel
15
Muscle & nerve
9
Journal of neuromuscular diseases
7
Muscle & Nerve
6
Neurology
6
Neurotherapeutics
5
Neurobiology of aging
4
Annals of neurology
4
Neurologic clinics
4
The Lancet. Neurology
3
JAMA neurology
3
Neuromuscular disorders : NMD
3
Neurotherapeutics : the journal of the American...
3
Trials
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Contemporary clinical trials
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Current opinion in neurology
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JAMA
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Journal of health economics and outcomes research
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Thema
72
Journal Article
29
Research Support, Non-U.S. Gov't
21
Randomized Controlled Trial
17
Duchenne muscular dystrophy
17
Multicenter Study
11
Research Support, N.I.H., Extramural
11
Review
10
Dystrophin
9
Clinical Trial, Phase III
8
Prednisone
8
VB0R961HZT
8
spinal muscular atrophy
7
Biomarkers
7
SMN2 protein, human
7
Survival of Motor Neuron 2 Protein
6
Clinical Trial, Phase II
5
Comparative Study
5
Pregnenediones
5
deflazacort
4
Anti-Inflammatory Agents
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Erscheinungszeitraum
113
2000-
2
1900-1999
Erscheinungsjahr(e)
Von:
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Sprache
100
Englisch
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