F. Buccella

发表

A harmonized and efficient clinical research environment would benefit patients and enhance European competitiveness. Commentary.

P. Pelicci, S. Boccia, A. Amato, 2017, Annali dell'Istituto superiore di sanita.

Evidence‐Based Consensus and Systematic Review on Reducing the Time to Diagnosis of Duchenne Muscular Dystrophy

S. Nelson, M. Hegde, A. Ferlini, 2019, The Journal of pediatrics.

226th ENMC International Workshop: Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy 20–22 January 2017, Heemskerk, The Netherlands

C. Szigyarto, F. Muntoni, A. Ferlini, 2018, Neuromuscular Disorders.

Duchenne muscular dystrophy and caregiver burden: a systematic review

Hanns Lochmüller, F. Buccella, J. Kirschner, 2018, Developmental medicine and child neurology.

Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30–31 January 2017

E. Mazzone, A. Mayhew, E. Mercuri, 2018, Neuromuscular Disorders.

Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry.

E. Mercuri, F. Muntoni, I. Desguerre, 2019, Journal of comparative effectiveness research.

Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA

H. Morizono, R. Finkel, Y. Hathout, 2017, Neuromuscular Disorders.

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

Christophe Béroud, Hanns Lochmüller, Petr Brabec, 2015, Human mutation.

Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database

K. Bushby, K. Flanigan, C. Béroud, 2017, Journal of neuromuscular diseases.

227th ENMC International Workshop: Finalizing a plan to guarantee quality in translational research for neuromuscular diseases Heemskerk, Netherlands, 10–11 February 2017

E. Clementi, M. Girgenrath, V. Dubowitz, 2017, Neuromuscular Disorders.

Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

E. Mercuri, F. Muntoni, I. Desguerre, 2020, Journal of comparative effectiveness research.

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

F. Muntoni, I. Desguerre, A. Kristensen, 2023, Journal of Neurology.

This Work Is Licensed under a Creative Commons Attribution 4.0 International License the Treat-nmd Dmd Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

K. Bushby, K. Flanigan, C. Béroud, 2022 .