Prospective measurement of quality of life in myotonic dystrophy type 1.
Stojan Z PericC HeatwoleE DurovicA KacarA NikolicI BastaA MarjanovicZ StevicD LavrnicV Rakocevic StojanovicPublished in: Acta neurologica Scandinavica (2017)
Individualized Neuromuscular Quality of Life questionnaire scores improved in our cohort of DM1 patients during a 6-year period. INQoL score did not correlate with progression of muscle weakness. This must be better understood before the selection of the instrument for use in trials to measure therapeutic benefit in DM1 patients.