Many of the internal organ systems of Drosophila melanogaster are functionally analogous
to those in vertebrates, including humans. Although humans and flies differ greatly in terms …

Fuchs endothelial corneal dystrophy (FECD) is a common cause for heritable visual loss in
the elderly. Since the first description of an association between FECD and common …

The Drosophila heart, also referred to as the dorsal vessel, pumps the insect blood, the
hemolymph. The bilateral heart primordia develop from the most dorsally located …

Myotonic dystrophy type 1 (DM1) is a chronically debilitating, rare genetic disease that
originates from an expansion of a noncoding CTG repeat in the dystrophia myotonica …

Highlights•Up to 34 drug candidates identified in the last few years.•Drug screening methods
available at all levels of complexity (computational to in vivo).•Translational research has …

Myotonic dystrophy type 1 (DM1), the most common muscular dystrophy affecting adults and
children, is a multi-systemic disorder affecting skeletal, cardiac, and smooth muscles as well …

Highlights•RNA medicine shows promise in DM1 therapeutics by targeting toxic CUG
expansions.•Restoring function of Muscleblind-like proteins is especially important in …

Abstract Myotonic dystrophy type 1 (DM1), the most common adult muscular dystrophy, is an
autosomal dominant disorder caused by an expansion of a (CTG) n tract within the 3 …

Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy in adults for which
there is currently no treatment. The pathogenesis of this autosomal dominant disorder is …

Pentamidine (PTM), an antiprotozoal agent used in clinics as pentamidine isethionate salt
(PTM-S), recently showed high potential also for the treatment of cancer and myotonic …