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

Andrew R FindlaySarah E RobinsonStephanie PoelkerMichelle SeiffertRocio BengoecheaConrad C Weihl

Published in: Annals of clinical and translational neurology (2022)

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.

Keyphrases

clinical trial
end stage renal disease
newly diagnosed
ejection fraction
chronic kidney disease
peritoneal dialysis
randomized controlled trial
risk assessment
current status
human health
phase iii