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1 . Academic Journal
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.
저자
by
Mercuri, Eugenio
;
Osorio, Andrés Nascimento
;
Muntoni, Francesco
;
Buccella, Filippo
;
Desguerre, Isabelle
, et al.
소스
Journal of Neurology
. Aug2023, Vol. 270 Issue 8, p3896-3913. 18p.
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7 . Academic Journal
Evidence-Based Consensus and Systematic Review on Reducing the Time to Diagnosis of Duchenne Muscular Dystrophy
저자
by
Aartsma-Rus, Annemieke
;
Hegde, Madhuri
;
Ben-Omran, Tawfeg
;
Buccella, Filippo
;
Ferlini, Alessandra
, et al.
소스
In
The Journal of Pediatrics
January 2019 204:305-313
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7 . Academic Journal
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
저자
by
Mercuri, Eugenio
;
Osorio, Andrés Nascimento
;
Muntoni, Francesco
;
Buccella, Filippo
;
Desguerre, Isabelle
, et al.
소스
Journal of Neurology
. 270(9):4583-4583
Scopus
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7 . Conference
Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA
저자
by
Bonnemann, Carsten
;
Boutin, Marc
;
Brais, Bernard
;
Buccella, Filippo
;
Burghes, Arthur
, et al.
소스
In
Neuromuscular Disorders
July 2017 27(7):693-701
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7 . Academic Journal
Effect of Ataluren on Age at Loss of Ambulation in Nonsense Mutation Duchenne Muscular Dystrophy: Observational Data from the STRIDE Registry.
저자
by
Werner, Christian
;
Delage, Abdallah
;
Buccella, Filippo
;
Desguerre, Isabelle
;
Muntoni, Francesco
, et al.
소스
Neuropediatrics
. 2019 Supplement2, Vol. 50, pS1-S55. 55p.
Scopus
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7 . Periodical
Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
저자
by
Mercuri, Eugenio
;
Muntoni, Francesco
;
Osorio, Andrés Nascimento
;
Tulinius, Már
;
Buccella, Filippo
, et al.
소스
Journal of Comparative Effectiveness Research; March 2020, Vol. 9 Issue: 5 p341-360, 20p
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7 . Periodical
Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry
저자
by
Muntoni, Francesco
;
Desguerre, Isabelle
;
Guglieri, Michela
;
Osorio, Andrés Nascimento
;
Kirschner, Janbernd
, et al.
소스
Journal of Comparative Effectiveness Research; August 2019, Vol. 8 Issue: 14 p1187-1200, 14p
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7 . Academic Journal
Duchenne muscular dystrophy and caregiver burden: a systematic review.
저자
by
Landfeldt, Erik
;
Edström, Josefin
;
Buccella, Filippo
;
Kirschner, Janbernd
;
Lochmüller, Hanns
.
소스
Developmental Medicine & Child Neurology
. Oct2018, Vol. 60 Issue 10, p987-996. 10p.
Scopus
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7 . Academic Journal
227th ENMC International Workshop:: Finalizing a plan to guarantee quality in translational research for neuromuscular diseases Heemskerk, Netherlands, 10–11 February 2017.
저자
by
Willmann, Raffaella
;
Buccella, Filippo
;
De Luca, Annamaria
;
Grounds, Miranda D.
.
소스
Neuromuscular Disorders
. Feb2018, Vol. 28 Issue 2, p185-192. 8p.
Full Text (Clinical Key)
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6 . Academic Journal
A harmonized and efficient clinical research environment would benefit patients and enhance European competitiveness.
저자
by
Amato, Antonino
;
Aringhieri, Eugenio
;
Boccia, Stefania
;
Buccella, Filippo
;
Gorini, Barbara
, et al.
소스
Annali dell'Istituto Superiore di Sanita; 2017, Vol. 53 Issue 2, p104-107, 4p
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