One of the challenges faced by doctors treating patients with myotonic dystrophy type 1 (DM1)—and drug developers designing clinical trials—is the broad difference in the way the disease manifests itself and progresses from patient to patient. MDF Scientific Advisory Committee member Dr. Guillaume Bassez, a neurologist at the Institut de Myologie in Paris, has identified subgroups of the DM population to help address this issue.
The OPTIMISTIC clinical evaluation of exercise training and cognitive behavioral therapy paradigms comes to publication.
Feasibility is demonstrated in mammalian models for a therapeutic strategy that increases MBNL by sequestering MBNL-suppressive miRs.
Insights on the mechanisms that underlie muscle atrophy in DM are provided by studies in a new mouse model.
The RNA binding protein, rbFOX1, competes with MBNL in binding to CCUGexp, but not CUGexp repeats, and thereby mitigates DM2.