Uncategorized Archives | International Rett Syndrome Foundation

December 12, 2025

Acadia Pharmaceuticals Announces FDA Approval of DAYBUE® STIX (trofinetide) for Oral Solution, a New Powder Formulation of Trofinetide for the Treatment of 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.

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.

Spotlight on Communication: An Interview with Dr. Theresa Bartolotta

For Theresa Bartolotta, Ph.D., CCC-SLP, good things have come through community. Bartolotta, a speech-language pathologist (SLP) with more than 40 years of clinical experience, discovered her passion for helping others […]

Advocating for YOU: Rare Disease Week on Capitol Hill

IRSF works tirelessly to advocate for you and your loved one with Rett syndrome. Our work takes many forms, our persistence is often unseen, and our successes are the result […]

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 […]

My Rett Ally: Revolutionizing Care Management for Rett Families

On a recent visit to the emergency room, it took just seconds for Samantha Brant to pull up a detailed, up-to-date medication list for her daughter, Macy. Macy lives with […]

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, […]

IRSF Blog