Taysha announced the FDA has granted Fast Track Designation (FTD) to TSHA-102 for Rett. FTD is designed to help treatments reach patients faster by expediting the review of therapies with potential to address unmet medical needs for a serious condition. Benefits of FTD to programs include early ...

New preclinical data after neonatal administration in wild-type mice showed no detectable impact on survival, neurobehavioral functions and overall health, suggesting TSHA-102, engineered with novel miRARE technology, avoided toxic overexpression of MeCP2 within cells already expressing MeCP2

What can heart rate tell us about Rett? A lot! In an RSRT-funded study, Emory researchers were able to associate Rett syndrome severity with variations in overnight heart rate. Using heart rate to understand symptom severity provides a new way to measure symptoms directly and objectively, and ...

David Keener, a PhD candidate in the Morningside Graduate School of Biomedical Sciences Interdisciplinary Graduate Program, has received a Ruth L. Kirschstein National Research Service Award Individual Predoctoral Fellowship from the National Institute for Neurological Diseases and Stroke. The ...