A new study points to the timing of MBNL-dependent RNA processing defects as a major factor in the pathogenesis of CDM.
Although fatigue represents a substantial burden in DM1, tools must be validated to assess its diverse contributing factors in order to develop clinical trial endpoints and effective therapies.
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 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.
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