A new study points to the timing of MBNL-dependent RNA processing defects as a major factor in the pathogenesis of CDM.
News
08/07/2017 - 10:53am
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.
07/06/2017 - 3:11pm
Preclinical animal efficacy data used to support the scientific rational for Ionis’ phase 1/2 clinical trial in DM1 was just published.
07/06/2017 - 3:02pm
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.
07/06/2017 - 2:53pm
A large retrospective study helps clarify genotype-cardiac phenotype relationships in adult DM1.
