International Rett Syndrome Foundation

It's almost #GivingTuesday and once again, we ask you to join us in giving hope to families all over the world with Rett syndrome. This year, every gift – whether through our donation page, on Facebook, through an event, or by creating a personal fundraising page – will be matched up to $100,000! You have the power to fund trailblazing research seeking treatments and cures, empowering family support, and increasing awareness of Rett in honor of someone you love. Learn more at https://lnkd.in/gD9B-tc6.

At IRSF, we are committed to transforming the lives of families living with Rett syndrome. We are grateful for your continued support of our mission, allowing us to make significant contributions in funding trailblazing research, empowering families, and advocating for families all over the world. As we look back on the incredible strides made in this past year, we are filled with hope and gratitude. Thank you for making a difference. Wishing you a healthy and joyful holiday!

Registration is now open for the Child Neurology Foundation's course for caregivers titled, “From Surviving to Thriving.” This course, running December 2-22, 2024, is intended to provide support for families with children with complex healthcare needs. During the three modules of this 100% virtual training, families will learn strategies to successfully manage the challenges of parenting a child with a neurologic condition so that they can protect the mental well-being of themselves and members of their family. This training is open to all parents or caregivers who have a child with a neurologic condition and the cost of the course is $10/participant. (If the cost causes a financial hardship, please reach out to programs@childneurologyfoundation.org). Learn more and register here: https://lnkd.in/eKdNigpV

The scientific community is invited to join us in Boston next June for the 2025 IRSF Rett Syndrome Scientific Meeting! The IRSF Rett Syndrome Scientific Meeting is the only annual, global, and comprehensive research meeting focused exclusively on Rett syndrome. During this 2.5-day meeting, attendees representing academia, industry, and governmental agencies share their latest research advances and discuss ways to leverage learning in the lab to be rapidly deployed in the clinic. Save the date to join scientists and researchers from around the world studying #Rettsyndrome and Rett-related areas. Look out for information on abstract submissions, registration, hotel accommodations, and more early next year.

We are excited to announce that the 2025 RettAway is set for July 9-12, 2025. Save the date to join families from around the world in Carlsbad Village, near San Diego, California, next summer! 🏝️⛱️🌞🌊 IRSF’s RettAway is a destination vacation for families to spend time together and build connections with other members of our Rett community. It's an exclusive opportunity for families to “learn by doing” that adventures can be pursued and enjoyed by all — no sitting on the sidelines here! We'll have more information about the planned activities, hotel room blocks, registration, and more soon! We can't wait to see you there!

A new Rett syndrome CME opportunity is now available at rettsyndrome.org/cme! Featuring a series of videos and other interactive activities, the free series "Unlocking Hope: A Journey Through Understanding and Supporting Individuals With Rett Syndrome" will help medical professionals gain in-depth knowledge on and strategies for optimizing the care of patients with Rett syndrome. These activities are provided by Clinical Care Options, in partnership with the International Rett Syndrome Foundation (IRSF), and supported by an educational grant from Acadia Pharmaceuticals Inc.

This morning, Neurogene Inc. provided an update on the trial participant who received NGN-401 at the high dose and began experiencing a serious adverse event (SAE). The patient is in critical condition, and Neurogene is asking the Rett community to send their “heartfelt thoughts to her family, friends, and the dedicated clinicians who are caring for her.” We join the Rett community in sending our thoughts and prayers to the patient and their family at this time. Neurogene has paused its high-dose cohort upon initial notification of the SAE and does not plan to enroll any further participants at the high-dose level. The FDA completed a review of the safety data for NGN-401 and allowed Neurogene to proceed with the Phase 1/2 trial using the 1E15 vg dose (low-dose cohort). You may read the full press release here: https://lnkd.in/gDvN68bX

Did you have a long diagnostic journey to receive your child’s #Rettsyndrome diagnosis? A new study from Kean University is looking to investigate the diagnostic process for Rett syndrome from parental perspectives, explore experiences with healthcare providers throughout this journey, and provide feedback to HCPs to improve the diagnosis process for Rett syndrome. Available to parents in the U.S. with children diagnosed in or after 2019. Learn more in the myRett Trial Finder tool here: https://lnkd.in/d4sW8RhV

In a corporate update, Taysha Gene Therapies today shared updates on the progress of their REVEAL phase 1/2 clinical trial of investigational gene therapy TSHA-102 for Rett syndrome. In the release, they shared that TSHA-102 was generally well tolerated and there were no serious adverse events or dose-limiting toxicities reported in the first 2 adolescent/adult patients and first pediatric patient in the high dose cohort. The IDMC reviewed clinical data from these participants and approved proceeding with enrollment. Taysha has also completed a positive RMAT Type B Meeting with the FDA. Read the full press release here: https://lnkd.in/gTAyNXHv

Twenty-five years ago, the Rett research community was rocked by a landmark discovery: mutations in the MECP2 gene caused Rett syndrome. In this year's edition of the Rett Gazette, we reflect on the significance of that discovery and how a genetic basis for Rett syndrome fundamentally changed how scientists and researchers have approached Rett since. We also give updates on the latest research and clinical trials, share in-depth stories of hope from our community, and celebrate all that we have accomplished—together—this past year. IRSF's annual Rett Gazette is arriving in mailboxes across the country RIGHT NOW! Learn more about this year's issue or request a mailed copy (US only) at https://lnkd.in/eq6GgcxK.