IRSF Blog

May 10, 2024

Neurogene Receives Australian HREC Approval for Trial

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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 […]

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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

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Clinical Trial Updates from Taysha & Anavex

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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 […]

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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 […]

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FDA Grants Trofinetide Priority Review

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In-Person Strollathons Are Back!

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IRSF Expands Center of Excellence Network

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

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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

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

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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 […]

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

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