There have been new discoveries in the way that congenital myotonic dystrophy (CDM) is inherited.
MDF is also happy to help you understand whether something is in a legitimate clinical trial, an approved therapy…or not.
The UK DM Patient Registry reports on disease burden for 556 patients with a confirmed diagnosis of DM1.
A new DM1 mouse model, with postnatal expression of expanded CUG repeat RNA in the brain, implicates reduced MBNL1 and MBNL2 in the staging of pathological and functional changes.
Epigenetic modifications upstream of an expanded DMPK allele may underlie the maternal bias in the inheritance of CDM.