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268th ENMC workshop - Genetic diagnosis, clinical classification, outcome measures, and biomarkers in Facioscapulohumeral Muscular Dystrophy (FSHD): Relevance for clinical trials.

Authors :
Montagnese F
de Valle K
Lemmers RJLF
Mul K
Dumonceaux J
Voermans N
Source :
Neuromuscular disorders : NMD [Neuromuscul Disord] 2023 May; Vol. 33 (5), pp. 447-462. Date of Electronic Publication: 2023 Apr 08.
Publication Year :
2023

Abstract

Competing Interests: Declaration of Competing Interest Federica Montagnese: has served as a paid consultant for DYNE Therapeutics. Katy de Valle: has received grant funding from FSHD Society USA and Fulcrum Therapeutics Nicol Voermans: is PI of the REACH trial of Fulcrum at the Radboud university medical center, Nijmegen, the Netherlands, and member of the steering committee of the trial. Financial compensation is paid to the hospital. She is the chair of the FSHD European Trial Network. Julie Dumonceaux: none Karlien Mul: has served as a paid consultant for Avidity Biosciences. Richard Lemmers: none

Details

Language :
English
ISSN :
1873-2364
Volume :
33
Issue :
5
Database :
MEDLINE
Journal :
Neuromuscular disorders : NMD
Publication Type :
Report
Accession number :
37099914
Full Text :
https://doi.org/10.1016/j.nmd.2023.04.005