Article
LGMDD1 natural history and phenotypic spectrum: Implications for clinical trials.
Annals of clinical and translational neurology - 1 Feb 2023
Findlay Andrew R, Robinson Sarah E, Poelker Stephanie, Seiffert Michelle, Bengoechea Rocio, Weihl Conrad C
Abstract excerpt
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 muscle testing and quantitative dynamometry data were used to estimate annualized rates of change. We also conducted a...
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