LGMDD1 natural history and phenotypic spectrum: Implications for clinical trials

ABSTRACT Objective To delineate the full phenotypic spectrum and characterize the natural history of limb girdle muscular dystrophy type D1 (LGMDD1). Methods We extracted age at clinical events of interest contributing to LGMDD1 disease burden via a systematic literature and chart review. Manual mus...

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Bibliographic Details
Main Authors: Andrew R. Findlay, Sarah E. Robinson, Stephanie Poelker, Michelle Seiffert, Rocio Bengoechea, Conrad C. Weihl
Format: Article
Language:English
Published: Wiley 2023-02-01
Series:Annals of Clinical and Translational Neurology
Online Access:https://doi.org/10.1002/acn3.51709