Category

Clinics

A First for Rett: FDA Approves Trofinetide for Treatment of Rett Syndrome!

Friday, March 10, 2023

This first-ever treatment is approved for individuals with Rett syndrome. (Updated March 19, 2023) Today, March 10, 2023, Acadia Pharmaceuticals announced that their investigational drug, Trofinetide, has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome. The drug, marketed under the name DAYBUE™, is expected to be commercially … Read More

Taysha Gene Therapies Provides Update on Expansion of TSHA-102 Study

Wednesday, February 1, 2023

READ TAYSHA’S LETTER TO THE RETT COMMUNITY DALLAS, Jan. 31, 2023 (GLOBE NEWSWIRE) — Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, clinical -stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic rare diseases of the central nervous system (CNS), today provided an update on the TSHA-120 program in giant axonal … Read More

IRSF Awards $4.4M in Rett Syndrome Research Grants

Tuesday, January 31, 2023

Our commitment to setting up the next wave of Rett breakthroughs continues with the largest annual investment in our Foundation’s history. By Dr. Dominique Pichard, IRSF Chief Science Officer We are entering new territory for Rett syndrome. Right now, the FDA is evaluating the first-ever potential treatment specifically targeting Rett syndrome, trofinetide. No potential treatment … Read More

Neurogene Announces First Rett Syndrome Gene Therapy Trial in U.S.

Monday, January 23, 2023

We’re excited to share that Neurogene announced today that the FDA approved their Investigational New Drug Application (IND) to launch a clinical trial for their gene therapy treatment NGN-401 in 2023 in pediatric females. This is the second gene therapy clinical study in Rett syndrome currently underway and the very first gene therapy clinical trial … Read More

Taysha Begins Recruitment for Gene Therapy Trial

Thursday, November 17, 2022

Next Huge Milestone for Rett The last 12 months have been an unprecedented time for Rett syndrome. In December 2021, Acadia Pharmaceuticals reported that the first (and only) Phase 3 clinical trial in Rett syndrome had positive results. As hopefully many of you know, Acadia submitted a New Drug Application to the FDA for approval … Read More

A FIRST FOR RETT: FDA approves Trofinetide for the treatment of Rett syndrome!
It’s Giving Tuesday - Go ALL IN for Rett!

A FIRST FOR RETT: FDA approves Trofinetide for the treatment of Rett syndrome!