A new literature review suggests meta-analysis of cognitive studies in DM1 is both feasible and can inform understanding of disease mechanisms and patient management.
It would be easy to conclude that insulin resistance and diabetes in myotonic dystrophy is a product of mis-splicing of INSR transcripts—but is that the full story?
Toxic RNA-mediated mis-splicing and Repeat-Associated Non-AUG (RAN) translation proteins are impacted by free MBNL1 levels and contribute towards CNS pathogenesis in DM2.
AMO Pharma released interim results for a Phase 2a safety and toxicity study of Tideglusib (also known as AMO-02) for adolescents and adults with congenital myotonic dystrophy.
New data is available on the frequency, progression and treatment of GI symptoms in DM1 and DM2.