A new publication finds a complex mechanism of action of furamidine in DM1 models.
Close monitoring of respiratory function reduces respiratory complications in DM1.
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.