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 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, […]
Taysha Gene Therapies Updates Community on Positive Initial Findings and Expansion of TSHA-102 Clinical Trial
Today, Taysha Gene Therapies made several exciting announcements about the progress of their REVEAL Phase 1/2 trial in Canada and the future study of their gene therapy, TSHA-102. To read […]
Acadia Pharmaceuticals Acquires International Rights to Trofinetide
BIG NEWS for our international Rett syndrome community! Today, Acadia Pharmaceuticals announced they have acquired the rights to make trofinetide available for the treatment of Rett syndrome across the globe. […]
Clinical Trial Updates from Taysha & Anavex
We are excited to share news from two pharmaceutical companies working on potential treatments and cures for Rett syndrome. Taysha Gene Therapies Taysha Gene Therapies shared that they are encouraged […]