Muscle MRI Findings in Childhood/Adult Onset Pompe Disease Correlate with Muscle Function

Figueroa-Bonaparte S, Segovia S, Llauger J, Belmonte I, Pedrosa I, Alejaldre A, Mayos M, Suárez-Cuartín G, Gallardo E, Illa I, Díaz-Manera J; Spanish Pompe Study Group. 
PLoS One. 2016. 6;11(10):e0163493.
The main objective of the study was to assess the use of muscle MRI in childhood/adult onset Pompe disease.
A good correlation is find between MFM-20 score and MRC (Muscle Research Council scale) and MRI-T1w scores.

PubMed link

Articles dans les revues scientifiques

Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial

Bertini E, Dessaud E, Mercuri E, Muntoni F, Kirschner J, Reid C, Lusakowska A, Comi GP, Cuisset JM, Abitbol JL, Scherrer B, Ducray PS, Buchbjerg J, Vianna E, van der Pol WL, Vuillerot C, Blaettler T, Fontoura P; Olesoxime SMA Phase 2 Study Investigators Lancet Neurol....

read more