A recent publication suggests that the DM field may be on the cusp of having a critically important molecular biomarker to facilitate decision-making in early-stage clinical trials.
For the first time, the 2016 MDF Annual Conference included a closed, professionals-only track focused on providing updates on basic scientific discovery and efforts focused on therapy development.
New studies in mouse models show that constitutive or acquired loss of Dmpk has no effects on skeletal or cardiac muscle function.
Since DM1-related loss of muscle mass confounds a common biomarker of kidney dysfunction, creatinine levels, serum cystatin C provides a superior measure of renal dysfunction for both patient management and clinical trials.
A recent study corroborated increased susceptibility to cancer in DM1, for women in particular, and linked the elevated risk to depressed levels of a tumor suppressor microRNA (miRNA).