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.

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Protocol for a phase II, monocentre, double-blind, placebo-controlled, cross-over trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2-4 (SPACE trial)

Stam M, Wadman RI, Wijngaarde CA, Bartels B, Asselman FL, Otto LAM, Goedee HS, Habets LE, de Groot JF, Schoenmakers MAGC, Cuppen I, van den Berg LH, van der Pol WL. BMJ Open. 2018. 30;8(7):e019932.  Protocol of the "SPACE" in wich MFM is used as an outcome measure....

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