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PubPharm (559)
1
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease : a phase III open-label extension study (ATB200-07)
enthalten in:
Journal of neurology
| 2024
von
Schoser, B.
|
Kishnani, P.
|
Bratkovic, D.
| +81
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2
Expert recommendations for magnetic resonance imaging of muscle disorders
enthalten in:
Radiologie (Heidelberg, Germany)
| 2024
von
Zeng, R.
|
Schlaeger, S.
|
Türk, M.
| +21
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3
Myasthenia Gravis - Update
enthalten in:
Fortschritte der Neurologie-Psychiatrie
| 2024
von
Gutschmidt, K.
|
Schoser, B.
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4
Minimal clinically important differences in six-minute walking distance in late-onset Pompe disease
enthalten in:
Orphanet journal of rare diseases
| 2024
von
Claeys, K.
|
Kushlaf, H.
|
Raza, S.
| +7
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5
Minimal clinically important differences in six-minute walking distance in late-onset Pompe disease
enthalten in:
Orphanet journal of rare diseases
| 2024
von
Claeys, K.
|
Kushlaf, H.
|
Raza, S.
| +7
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6
Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease : an open-label phase I/II study (ATB200-02)
enthalten in:
Journal of neurology
| 2024
von
Byrne, B.
|
Schoser, B.
|
Kishnani, P.
| +17
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7
Therapeutic resistance training: proposal for an algorithm-based approach
enthalten in:
Translational exercise biomedicine
| 2024
von
Weber, F.
|
Stark, C.
|
Bloch, W.
| +12
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8
Correction to : Analysis of muscle magnetic resonance imaging of a large cohort of patient with VCP‑mediated disease reveals characteristic features useful for diagnosis
enthalten in:
Journal of neurology
| 2024
von
Esteller, D.
|
Schiava, M.
|
Verdú-Díaz, J.
| +63
ErratumFor: J Neurol. 2023 Dec;270(12):5849-5865. - PMID 37603075
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9
Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa : Meaningful change analyses from the Phase 3 COMET trial
enthalten in:
Molecular genetics and metabolism
| 2024
von
Toscano, A.
|
Pollissard, L.
|
Msihid, J.
| +10
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10
Correction to: Analysis of muscle magnetic resonance imaging of a large cohort of patient with VCP‑mediated disease reveals characteristic features useful for diagnosis
enthalten in:
Journal of neurology
| 2024
von
Esteller, D.
|
Schiava, M.
|
Verdú-Díaz, J.
| +63
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Der Nervenarzt
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Molecular genetics and metabolism
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Brain : a journal of neurology
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Current opinion in neurology
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Neurology
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American journal of human genetics
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Acta neuropathologica
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Enzyme replacement therapy
20
EC 3.2.1.20
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Glycogen storage disease type 2
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alpha-Glucosidases
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GAA protein, human
13
Multicenter Study
13
Muscle Proteins
13
Research Support, N.I.H., Extramural
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Creatine Kinase
12
RNA-Binding Proteins
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Randomized Controlled Trial
11
Myotonic dystrophy
10
EC 2.7.3.2
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Myotonic dystrophy type 2
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