Clinical trial preparedness in facioscapulohumeral muscular dystrophy: Clinical, tissue, and imaging outcome measures 29–30 May 2015, Rochester, New York

Rabi Tawil; George W Padberg; Dennis W Shaw; Silvère M van der Maarel; Stephen J Tapscott; FSHD Workshop Participants

doi:10.1016/j.nmd.2015.10.005

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Clinical trial preparedness in facioscapulohumeral muscular dystrophy: Clinical, tissue, and imaging outcome measures 29–30 May 2015, Rochester, New York

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Clinical trial preparedness in facioscapulohumeral muscular dystrophy: Clinical, tissue, and imaging outcome measures 29–30 May 2015, Rochester, New York

Rabi Tawil, George W Padberg, Dennis W Shaw, Silvère M van der Maarel, Stephen J Tapscott and FSHD Workshop Participants

Neuromuscular disorders : NMD, Vol.26(2), pp.181-186

02/2016

DOI: 10.1016/j.nmd.2015.10.005

PMID: 26627872

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Abstract

•Many national registries now exist facilitating patient access to trials.•New functional and patient reported outcome measure is being validated.•More research is required to establish reliable tissue and serum biomarkers.•Longitudinal muscle MRI studies suggest that imaging is a useful biomarker in FSHD.•Electrical impedance myography is reliable in certain muscle groups.

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Title: Subtitle: Clinical trial preparedness in facioscapulohumeral muscular dystrophy: Clinical, tissue, and imaging outcome measures 29–30 May 2015, Rochester, New York
Creators: Rabi Tawil - Department of Neurology, University of Rochester Medical Center, Rochester, NY, USA
George W Padberg - Department of Neurology, Radboud University Medical Center, Nijmegen, The Netherlands
Dennis W Shaw - Department of Radiology, Seattle Children's Hospital, Seattle, WA, USA
Silvère M van der Maarel - Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands
Stephen J Tapscott - Division of Human Biology, Fred Hutchinson Cancer Research Center, Seattle, WA, USA
FSHD Workshop Participants
Contributors: Katherine D Mathews (Contributor) - University of Iowa, Stead Family Department of Pediatrics
Resource Type: Journal article
Publication Details: Neuromuscular disorders : NMD, Vol.26(2), pp.181-186
DOI: 10.1016/j.nmd.2015.10.005
PMID: 26627872
NLM abbreviation: Neuromuscul Disord
ISSN: 0960-8966
eISSN: 1873-2364
Publisher: Elsevier B.V
Grant note: FSHD Global, Australia FSHD Stichting, The Netherlands FSH Society, United States
Language: English
Date published: 02/2016
Academic Unit: Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
Record Identifier: 9984020705002771

Clinical trial preparedness in facioscapulohumeral muscular dystrophy: Clinical, tissue, and imaging outcome measures 29–30 May 2015, Rochester, New York

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