LGMDD1 natural history and phenotypic spectrum: Implications for clinical trials.

Andrew R Findlay,Sarah E Robinson,Stephanie Poelker,Michelle Seiffert,Rocio Bengoechea,Conrad C Weihl

Annals of clinical and translational neurology（2023）

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摘要

This study is the largest description of LGMDD1 patients to date and highlights potential genotype-dependent differences that need to be verified prospectively. Future clinical trials will need to account for variability in these key phenotypic features when selecting outcome measures and enrolling patients.

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clinical,phenotypic spectrum

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