Myotonic Dystrophy and the Brain: Causes, Effects and Treatment

Presented during Myotonic's Friday Afternoon Webinar Series.

DM can cause mental fatigue, daytime sleepiness, forgetfulness, confusion or “brain fog”, all related to altered brain activity. However, there is some good news – the number of nerve cells in the brains of people with DM is nearly normal. The nerves do, though, have molecular and functional abnormalities caused by the underlying DM genetic change. These features suggest that brain function in DM might be improved with treatment, which some drug trials have already started to address. Many other potential treatments are in the pipeline. We are eager to work with the DM community to prepare for these upcoming trials by validating methods that accurately measure treatment effects.

About the Presenters:
Dr. John Day is a professor of neurology, pediatrics and pathology at Stanford University, where is the Diirector of Neuromuscular Medicine. Dr. Day has more than 30 years of experience diagnosing and caring for patients of all ages with DM. 

Dr. Jacinda Sampson is a clinical associate professor at Stanford University Hospital and Clinics. She is a neuromuscular and neurogenetics specialist who has been caring for patients and families with DM for over 12 years. 

Dr. Gayle Deutsch is a Clinical Professor (Affiliated) in the Department of Neurology and Neurological Sciences. She is the lead neuropsychologist for the Stanford Neuropsychology Service.

Dr. Tahereh Kamali is a postdoctoral fellow that joined Stanford in September 2019. Her research interests primarily lie in the design of new machine learning techniques for healthcare and developing clinical decision support systems to achieve accurate and robust prediction particularly in case of having partially-labeled training data.