Clinical and healthcare burden of myotonic dystrophy type 1 (DM1) in the United States: a claims-based study

DM1 imposes significant clinical and economic burden, marked by extensive organ involvement and variable healthcare costs. As any patient may progress to costly cardiac or pulmonary complications, early proactive management is critical. The complexity and heterogeneity of DM1 highlight ongoing unmet needs and the importance of developing effective treatments.