Northwest Rett Syndrome Association

Today, Neurogene Inc. announced the completion of dosing in the Embolden registrational trial of investigational gene therapy, NGN-401.

Due to strong demand, 25 participants have been dosed, exceeding the original target of 20, and NGN-401 has been generally well tolerated, with no cases of HLH at the 1E15 vg dose. Neurogene expects to share updated interim Phase 1/2 data in mid-2026 and topline data from Embolden in the second half of 2027.

We look forward to welcoming the Neurogene team to the IRSF Rett Syndrome Scientific Meeting and ASCEND National Summit later this month.

Read the full press release here: https://ir.neurogene.com/news-releases/news-release-details/neurogene-announces-successful-completion-dosing-emboldentm

Support Safer Spaces: https://bit.ly/4rGYm7s

Home modifications can make daily routines more secure and accessible for individuals with Rett syndrome. Changes like accessible bathrooms, ramps, lift systems, and adaptable furniture can create safer pathways through everyday life.

Are you interested in our next webinar in our Connection Course Series?! There are only TWO days left to register for The Sensory Connection! Don’t miss this opportunity to take the newly revamped course with AHA, Inc. with Gina Taylor, MS, OT, HPCS✨

This two-day online course applies sensory integration theory, reviews neuroanatomy, and covers horse selection and use of the environment to address sensory processing challenges. Participants will gain an understanding of treatment for children with sensory processing challenges using equine movement as a treatment tool and will engage in hands-on activities and clinical reasoning as part of the course to ensure learning is carried over to the therapists’ home practice.

Spots are limited, so head to the comments or the link in our bio for registration info before Monday!📖🐴💻

Hello everyone, here is the schedule for the Rett Adventure, also the cutoff for the Best Western and La Quinta room block is 6/15 so please book asap. We can’t wait to see you all!

Want to get involved with Disability EmpowHer Network? Join us for a webinar on Zoom on June 17 at 12pm ET to discuss volunteer opportunities. Sign up here: https://docs.google.com/forms/d/e/1FAIpQLScyNWJfgkL6pSfKI2rJx5x8njHk8WwPKThAW0i1OBeVegA2zw/viewform

ID: A graphic with photos of diverse disabled girls and nonbinary youth. Text reads Volunteers wanted. Ready to make a real difference? Become a volunteer for DEN and help create a positive impact in the community!

Start with accessibility in mind. Accessibility benefits EVERYONE. Not just a small population.

Lundbeck is hosting a 1-hour, virtual webinar for families and caregivers navigating severe and rare developmental epilepsies—often referred to as Developmental and Epileptic Encephalopathy (DEE). This session will provide information about DEE and Lundbeck’s ongoing clinical research efforts in this space, including:

• An overview of DEE
• Overview of ongoing, actively enrolling Phase 3 clinical studies
• Responses to frequently asked questions

To register: https://zoom.us/webinar/register/5317769790342/WN_ceq3_KCVTW6qhuqCkhQmxQ

For more information, please reach out to [email protected].