Long-term treatment with Daybue (trofinetide) led to continual improvements among children and young adults with Rett syndrome in…
Articles by Marisa Wexler, MS
When clinicians have to decide whether to recommend invasive ventilation for adolescents with Rett syndrome, parental and familial support…
The U.S. Food and Drug Administration (FDA) has given a regenerative medicine advanced therapy (RMAT) designation to TSHA-102, an experimental…
The Rett Syndrome Research Trust (RSRT) is teaming up with partners in industry and academia to get ready for…
The first girl with Rett syndrome has been given the experimental gene therapy TSHA-102 in a clinical trial, according…
Treatment with Anavex 2-73 (blarcamesine) led to improvements in behavior among children with Rett syndrome in a Phase 2/3…
Rett syndrome is marked by unusually low levels of LAT1, a protein that’s needed to move amino acids —…
Electrically stimulating a specific region of the brain led to improvements in memory in a mouse model of Rett…
Treatment with a modified version of a signaling molecule called NGF improved survival and normalized behavior in a mouse model…
A second adult with Rett syndrome has been given the experimental gene therapy TSHA-102 in an ongoing clinical trial,…