The underlying cause of LCHADD retinopathy is not fully understood. This presentation will look at the research that identifies the cell in the eye that is initially affected and characteristics associated with vision loss. Join Dr. Gillingham as she discusses early data on the natural history study and some new pre-clinical models to test novel treatments for LCHADD retinopathy.
Siblings of children with disabilities play important roles in their families, and often experience similar concerns and opportunities as parents. This session will present...
Talking points include: What are Standards of Care and why does the Mito community need such standards? Review the MMS’s Standards of Care for...
When a genetic variant is shared by only a handful of individuals worldwide, what does it mean for diagnosis, treatment, and research? In this...
