LAMA2-related muscular dystrophy: Natural history of a large pediatric cohort.

Alberto Andrea ZambonDeborah RidoutMarion MainRachael MeinRahul PhadkeFrancesco MuntoniAnna Sarkozy

Published in: Annals of clinical and translational neurology (2020)

This retrospective longitudinal study provides long-term natural history of LAMA2-RD. This will help management and identification of key milestones of disease progression that could be considered for future therapeutic intervention.

Keyphrases

muscular dystrophy
duchenne muscular dystrophy
randomized controlled trial
current status
cross sectional
bioinformatics analysis
drug induced