Preclinical animal efficacy data used to support the scientific rational for Ionis’ phase 1/2 clinical trial in DM1 was just published.
New fly models show a DM2 phenotype at least as severe as DM1 and may provide a platform for studies of genetic modifiers and candidate therapy screening in DM.
A large retrospective study helps clarify genotype-cardiac phenotype relationships in adult DM1.
A recent review article makes the case that DM is a brain disease and that better understanding of and treatment strategies for the neurological consequences of DM are essential.
MDF staff recently attended the 2017 annual meeting of the American Academy of Neurology, in Boston, MA. Here are highlights from that meeting.
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.
Sitemap | User Agreement
© Myotonic Dystrophy Foundation. All rights reserved.