Clinical correlations and long-term follow-up in 100 patients with sarcoglycanopathies.
R Guimarães-CostaGorka Fernández-EulateK WahbiF LeturcqE MalfattiA BehinS Leonard-LouisI DesguerreC BarneriasM C NouguesA IsapofB Estournet-MathiaudS Quijano-RoyA FayssoilD OrlikowskiB FaurouxI RichardC SempliciniN B RomeroG QuerinB EymardPascal LaforêtT StojkovicPublished in: European journal of neurology (2020)
This large multicentric series delineates the clinical spectrum of patients with sarcoglycanopathies. Age at disease onset is an independent predictor of severity of disease and LoA, and should be taken into account in future clinical trials.