Aware That We’re Rare: The Power of Community in Rare Diseases
As rare disease advocates gathered for Rare Disease Week 2025, they reinforced a powerful truth: when we come together, our voices are impossible to ignore.
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.
IRSF Leadership Update
After a 7-year tenure, Melissa Kennedy stepped down as CEO at the end of 2024. Board member and Rett mom, Leslie Mehta, will act as interim CEO as a national search for a new leader commences.
Raising a Hand: A Journey of Healing, Hope, and Friendship
Inconceivable. That’s the word that photographer Dave Clements uses to describe the emotion swelling in his stomach when thinking back to March 9, 2003.
Update on Patient SAE & Discontinuation of High Dose Protocol (Community Letter)
Update on Patient SAE (Community Letter)
Neurogene Provides Update on NGN-401 Gene Therapy Clinical Trial for Rett Syndrome
Taysha Gene Therapies Reports Third Quarter 2024 Financial Results and Provides Corporate Update
Neurogene Community Letter
Neurogene Reports Positive Interim Efficacy Data from First Four Low-Dose Pediatric Participants in NGN-401 Gene Therapy Clinical Trial for Rett Syndrome
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.