Category

Family Empowerment

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

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

Flu, COVID, & RSV: A Message to our Rett Community

Thursday, December 8, 2022

By Dr. Tim Benke, IRSF Medical Advisor As many of you know, the United States is currently facing the triple impact of flu, RSV, and COVID-19. These respiratory illnesses are currently circulating at levels higher than usual for this time of year, creating a surge of patients seeking care in clinical offices, emergency rooms, and … 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

Your Voice Matters to the FDA & Pharmaceutical Industry

Thursday, October 13, 2022

By Dr. Dominique Pichard, IRSF CSO – The patient voice has increasingly become a focus for the Food and Drug Administration (FDA) in the drug development process. Thanks to years of advocacy from patients and caregivers, the FDA has developed mechanisms to hear the patient voice and has encouraged pharmaceutical companies to increasingly include the … Read More