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Disease burden of myotonic dystrophy type 1.

Erik LandfeldtNikoletta NikolenkoCecilia Jimenez-MorenoSarah CummingDarren G MoncktonGrainne GormanChris TurnerHanns Lochmüller
Published in: Journal of neurology (2019)
We show that DM1 is associated with a substantial disease burden resulting in impairment across many different domains of patients' lives, emphasizing the need for a holistic approach to medical management. Our results also show that the INQoL records relevant information about patients with DM1, but that further investigation of the psychometric properties of the scale is needed for meaningful interpretation of instrument scores.
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