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PubPharm (35)
1
Correction to : Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis
enthalten in:
Journal of neurology
| 2023
von
Mercuri, E.
|
Osorio, A.
|
Muntoni, F.
| +14
ErratumFor: J Neurol. 2023 Aug;270(8):3896-3913. - PMID 37115359
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2
Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022) : 2022 interim analysis
enthalten in:
Journal of neurology
| 2023
von
Mercuri, E.
|
Osorio, A.
|
Muntoni, F.
| +14
ErratumIn: J Neurol. 2023 Sep;270(9):4583. - PMID 37460854
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3
Correction to: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis
enthalten in:
Journal of neurology
| 2023
von
Mercuri, E.
|
Osorio, A.
|
Muntoni, F.
| +13
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4
Correction to: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis
enthalten in:
Journal of neurology
| 2023
von
Mercuri, E.
|
Osorio, A.
|
Muntoni, F.
| +13
Wird geladen...
5
Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis
enthalten in:
Journal of neurology
| 2023
von
Mercuri, E.
|
Osorio, A.
|
Muntoni, F.
| +13
Wird geladen...
6
Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis
enthalten in:
Journal of neurology
| 2023
von
Mercuri, E.
|
Osorio, A.
|
Muntoni, F.
| +13
Wird geladen...
7
Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis
enthalten in:
Journal of neurology
| 2023
von
Mercuri, E.
|
Osorio, A.
|
Muntoni, F.
| +13
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8
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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9
Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1
enthalten in:
Journal of neuromuscular diseases
| 2023
von
McDonald, C.
|
Mayer, O.
|
Hor, K.
| +12
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10
Natural History and Real-World Data in Rare Diseases : Applications, Limitations, and Future Perspectives
enthalten in:
Journal of clinical pharmacology
| 2022
von
Liu, J.
|
Barrett, J.
|
Leonardi, E.
| +4
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6
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Thema
17
Journal Article
12
Research Support, Non-U.S. Gov't
7
Codon, Nonsense
6
Duchenne muscular dystrophy
6
K16AME9I3V
6
STRIDE Registry
6
ataluren
5
Oxadiazoles
5
Prednisone
5
VB0R961HZT
5
nonsense mutation Duchenne muscular dystrophy
4
Ataluren
4
Effectiveness
4
Nonsense mutation Duchenne muscular dystrophy
4
Safety
4
deflazacort
4
efficacy
3
9PHQ9Y1OLM
3
Dystrophin
3
Multicenter Study
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Erscheinungszeitraum
31
2000-
4
1900-1999
Erscheinungsjahr(e)
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26
Englisch
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