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.
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 Expansion of Gene Therapy Clinical Trial
Today, Neurogene announced plans to expand and more rapidly enroll patients their Phase 1/2 clinical trial of investigational gene therapy NGN-401. For the full press release, click here. Key Facts: […]
Taysha Gene Therapies Adolescent & Adult Study Expanded to U.S.
Today, Taysha Gene Therapies shared several exciting updates on their REVEAL Phase 1/2 clinical trial of investigational gene therapy TSHA-102: The study will now expand to include adolescent and adult […]
Taysha Gene Therapies Doses First Pediatric Patient in U.S. Gene Therapy Clinical Trial
Taysha Gene Therapies today announced the dosing of the first pediatric patient in their REVEAL Phase 1/2 trial of investigational gene therapy TSHA-102. For Taysha’s full press release, click here. […]
Neurogene Announces First Pediatric Patients Dosed in US Gene Therapy Clinical Trial
We’re excited to share that today Neurogene announced that two pediatric patients have been dosed in their Phase 1/2 trial of investigational gene therapy NGN-401. For Neurogene’s full press release, […]