Open-Label Trial of Recombinant Human Insulin-like Growth Factor 1/Recombinant Human Insulin-like Growth Factor Binding Protein 3 in Myotonic Dystrophy Type 1

Abstract
Myotonic dystrophy type 1 (DM1) is a progressive multisystem degenerative disorder caused by expansion of a CTG repeat in the dystrophia myotonica protein kinase (DMPK) gene.1 Currently there is no known treatment capable of modifying the progressive myopathy of DM1.