IRSF Blog

March 24, 2025

Neurogene Reports Fourth Quarter and Full Year 2024 Financial Results and Highlights Recent Updates

Meghan Cordeiro

Share this post

More Recent News

Life After Rett: Finding Hope and Connection Beyond Loss

For parents facing the unthinkable loss of a child to Rett syndrome, a Facebook group called Life After Rett offers understanding, connection, and the reminder that no one grieves alone.

The Conversation Continues: Celebrating Five Years of The Rett Syndrome Communication Guidelines

Five years after its release, the Rett Syndrome Communication Guidelines continue to inspire a global movement in care and collaboration. What began as a set of best practices has grown into The Hive—a vibrant professional network—and has shaped graduate programs nationwide, ensuring individuals with Rett syndrome receive care that’s informed, compassionate, and connected.

More Than Rare: Understanding the Male Rett Spectrum

New research is breaking down outdated assumptions and bringing male experiences into full view.

Summer Is Here! Tips & Tools to Help

Summer may bring a shift in routine, but with the right support, it can also be a time for growth, connection, and a little more breathing room.

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.

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.

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.

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.

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, […]

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. […]

RettAway Reflections

By Samantha Brant, Family & Community Engagement Manager, and mom to Macy As I returned home from this year’s RettAway at Morgan’s Inspiration Island, my heart swelled with not only […]

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 […]

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 […]

Neurogene Announces First Rett Syndrome Gene Therapy Trial in U.S.

We’re excited to share that Neurogene announced today that the FDA approved their Investigational New Drug Application (IND) to launch a clinical trial for their gene therapy treatment NGN-401 in […]

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 […]

Taysha Begins Recruitment for Gene Therapy Trial

Next Huge Milestone for Rett The last 12 months have been an unprecedented time for Rett syndrome. In December 2021, Acadia Pharmaceuticals reported that the first (and only) Phase 3 […]

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 […]

FDA Grants Trofinetide Priority Review

ACADIA has shared the exciting news that the US Food and Drug Administration (FDA) has reviewed the documents received as part of the New Drug Application (NDA) for trofinetide, and […]

In-Person Strollathons Are Back!

In September IRSF returns with our in-person Strollathons! These nationwide fundraisers bring together local communities to go ALL IN for treatments and a cure for Rett syndrome. Since 2004, these […]

IRSF Expands Center of Excellence Network

The International Rett Syndrome Foundation is expanding our community’s access to best-in-class care by adding four Rett syndrome clinics to our Center of Excellence network. This expansion helps to fulfill […]

The Summit of Hope and Healing: ASCEND 2022 Rett Syndrome National Summit

By Katie Busch, IRSF Program Coordinator – During the last week of April 2022, the International Rett Syndrome Foundation welcomed more than 500 members of the Rett community to the […]

Accelerating Discoveries in Rett Research: 2022 IRSF Rett Syndrome Scientific Meeting

In April 2022, the International Rett Syndrome Foundation hosted the IRSF Rett Syndrome Scientific Meeting in advance of the foundation’s first in-person family summit in 6 years. The 2-day meeting […]

Taysha Rett Syndrome Community Update

From IRSF: 2022 is a big year for Rett syndrome: the return of an in-person Family Summit, a successful EL-PFDD meeting to share the voice of the community with the […]

IRSF Sets Up Next Wave of Rett Breakthroughs with $2.3 M Research Investment

Dr. Dominique Pichard, IRSF Chief Science Officer Get ready for the next wave of research targeting treatments and cures for Rett syndrome. IRSF is making it happen. “Because Rett syndrome […]

Positive Top-line Results for Phase 3 Trofinetide Trial

Melissa Kennedy, Chief Executive Officer On December 6, the Rett syndrome community received some very encouraging news: Acadia Pharmaceuticals reported that top-line results for its Lavender Phase 3 clinical trial for […]

Help Unlock Treatments & Cures for Rett with IRSF’s Rett Syndrome Registry

Your family can be the key that researchers and industry need to create a world without Rett. And the International Rett Syndrome Foundation (IRSF) is making it easier than ever […]

International Rett Syndrome Foundation’s New Center of Excellence Network

The International Rett Syndrome Foundation is committed to advancing and expanding our community’s access to best-in-class Rett syndrome care. Toward that end, we are designating 15 partner clinics across the […]

Kelsey & Toyua: Friendship & Hope Just 20 Minutes Away

Kelsey Clark felt alone and isolated when her five-year-old daughter Charlee was diagnosed with Rett syndrome in April 2019. Kelsey, her partner Jordan, Charlee, and their two-year-old son Bennett live […]

Board of Directors Welcomes Alan Shukovsky —The Father of a Boy Living With Rett

When Alan Shukovsky and his wife, Eva, were getting married, their rabbi told them that it was really important for their family to find a personal mission—a cause—they could support […]