Up to nearly half of children and adults with Rett syndrome in the U.S. have digestive…
Articles by Margarida Maia, PhD
A Phase 1/2 clinical trial has been cleared to infuse NGN-401, a one-time investigational gene therapy, to a third girl…
Effective communication is a top concern for people affected by Rett syndrome or related disorders, a U.S. caregiver survey…
Taysha Gene Therapies has received the go-ahead to continue clinical testing of TSHA-102, its gene therapy for Rett…
A pharmaceutical company in Italy is teaming up with scientists at the Sapienza University of Rome to find out if…
Anavex Life Sciences has completed dosing all 92 girls with Rett syndrome taking part in a clinical trial testing…
TSHA-102, an experimental gene therapy by Taysha Gene Therapies, can increase the levels of MeCP2 — a protein that’s…
Scientists may have discovered promising treatments for Rett syndrome in two small molecules, called KW-2449 and VPA, each seen…
NGN-401, a new gene therapy in the pipeline from Neurogene, extended the lifespan and reduced the disease burden in…
A mosaic mutation — one that is present in only some body cells — causing Rett syndrome was found…