Taysha Gene Therapies Announces Pivotal Part B Trial Design: A Promising Step Forward for the Rett Community
Positive clinical data from part A of Taysha Gene Therapies’ Phase 1/2 REVEAL trial of TSHA-102 support plans for a pivotal study with primary endpoints based on developmental milestones from the Rett Syndrome Natural History Study.
IRSF Expands Scientific Advisory Board with Three Leading Experts
Renowned leaders in neuroscience, gene therapy, and translational research join IRSF’s Scientific Advisory Board to help accelerate bold investments in Rett syndrome treatments and cures.
Pioneering Progress in Rett Research: IRSF Awards Nearly $2M to Fuel Bold New Science
Dr. Nupur Garg, IRSF’s VP of Research, shares how nearly $2 million in new grants reflect IRSF’s bold strategy to drive progress across the Rett research pipeline—and bring hope for meaningful change.
IRSF Awards Nearly $2 Million in Grants to Advance Rett Research
This funding supports seven groundbreaking projects investigating Rett syndrome’s pathology and potential new treatments.
DAYBUE in the Real World
Answers to your most frequently asked questions and resources to find out more about the first-ever FDA-approved treatment of Rett syndrome.
Transforming the Landscape of Rett Research
The International Rett Syndrome Foundation (IRSF) hosted its annual scientific meeting in Westminster, Colorado on June 18-19, 2024, as part of its ASCEND 2024 Rett Syndrome National Summit. By Mackenzie […]
Center of Excellence Clinic Network Expanded
Three additional clinics recognized for best-in-class Rett syndrome care.
IRSF Announces Expansion of Scientific Leadership
The new leadership appointments include a key promotion and new hire to advance research and clinical development for Rett syndrome.
Neurogene Announces Locations & Opens Enrollment for US Gene Therapy Clinical Trial
We’re excited to share that today Neurogene announced the locations and began enrolling for the first U.S. Phase 1/2 clinical trial of their investigational gene therapy, NGN-401. Enrollment at Texas […]
A First for Rett: FDA Approves Trofinetide for Treatment of Rett Syndrome!
This first-ever treatment is approved for individuals with Rett syndrome. (Updated March 19, 2023) On March 10, 2023, Acadia Pharmaceuticals announced that their investigational drug, Trofinetide, has been approved by […]
Flu, COVID, & RSV: A Message to our Rett Community
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 […]
Your Voice Matters to the FDA & Pharmaceutical Industry
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 […]