Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints
Abstract Objective Limb girdle muscular dystrophy type R9 (LGMD R9) is an autosomal recessive muscle disease for which there is currently no causative treatment. The development of putative therapies requires sensitive outcome measures for clinical trials in this slowly progressing condition. This s...
Main Authors: | Alexander P. Murphy, Jasper Morrow, Julia R. Dahlqvist, Tanya Stojkovic, Tracey A. Willis, Christopher D. J. Sinclair, Stephen Wastling, Tarek Yousry, Michael S. Hanna, Meredith K. James, Anna Mayhew, Michelle Eagle, Laurence E. Lee, Jean‐Yves Hogrel, Pierre G. Carlier, John S. Thornton, John Vissing, Kieren G. Hollingsworth, Volker Straub |
---|---|
Format: | Article |
Language: | English |
Published: |
Wiley
2019-06-01
|
Series: | Annals of Clinical and Translational Neurology |
Online Access: | https://doi.org/10.1002/acn3.774 |
Similar Items
-
Quantitative magnetic resonance imaging in limb-girdle muscular dystrophy 2I: a multinational cross-sectional study.
by: Tracey A Willis, et al.
Published: (2014-01-01) -
Quantitative muscle MRI as an assessment tool for monitoring disease progression in LGMD2I: a multicentre longitudinal study.
by: Tracey A Willis, et al.
Published: (2013-01-01) -
Upper Limb Evaluation in Duchenne Muscular Dystrophy: Fat-Water Quantification by MRI, Muscle Force and Function Define Endpoints for Clinical Trials.
by: Valeria Ricotti, et al.
Published: (2016-01-01) -
A natural history study in Limb Girdle Muscular Dystrophy 2I : magnetic resonance imaging, spectroscopy and physical outcome measures
by: Willis, Tracey Anne
Published: (2012) -
Global FKRP Registry: observations in more than 300 patients with Limb Girdle Muscular Dystrophy R9
by: Lindsay B. Murphy, et al.
Published: (2020-05-01)