Very Low Residual Dystrophin Quantity Is Associated with Milder Dystrophinopathy.

Yvan de FeraudyRabah Ben YaouKarim WahbiCaroline StalensAmalia StantzouVincent LaugelIsabelle Desguerrenull nullLaurent ServaisFrance LeturcqHelge Amthor

Published in: Annals of neurology (2020)

Very low residual dystrophin protein quantity can cause a shift in disease phenotype from DMD toward BMD. ANN NEUROL 2021;89:280-292.

Keyphrases

duchenne muscular dystrophy
muscular dystrophy
binding protein
amino acid
small molecule