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.
A new review article assesses the current status of and recommends future directions for brain imaging studies in DM1.
Given the brain manifestations of DM1, how aware are most patients of the impact and progression of their disease?
Learn more about Dr. Ami Mankodi, principal investigator at the National Institutes of Health’s (NIH) National Institute of Neurological Disorders and Stroke (NINDS) in Bethesda, Maryland. Dr. Mankodi has been involved in research that has helped shape a fundamental biologic and molecular understanding of myotonic dystrophy (DM).
A novel redirection of CRISPR/Cas9 technology addresses toxic RNA, rather than the genome, as a potential therapy for DM1 and DM2.
Understanding cardiac and other DM risk factors and planning for the known complications of DM that may affect you can help protect and maintain your quality of life and that of your loved ones.