It was a pleasure to attend the International Rett Syndrome Foundation (IRSF) scientific meeting held in Nashville June 5-7, 2023. The meeting consisted of both poster and oral presentation sessions and breakout discussions focused on basic, translational, and clinical work on Rett syndrome. Between sessions, attendees had ample time to connect, break down barriers, and forge new partnerships to advance toward treatments and a cure for Rett syndrome.
O.R.S.A. was well represented and acknowledged as an advocacy partner during the 3-day meeting. Our very own Canadian researcher from the Hospital for Sick Children, Dr. James Ellis, (2023 Hope Fund Grant Recipient) presented his research and acknowledge the funding contributions made by O.R.S.A. to support his work.
Valuable knowledge was gained, and connections were made with clinicians, researchers and pharmaceutical companies. Face-to-face meeting with the representatives from both Taysha Gene Therapies and Acadia Pharmaceuticals Inc has energized our commitment to our community as new promising therapies become available. We will continue our mission to build healthy tomorrow by investing efforts in advocating for treatments to be available in Canada.
As you may have heard, the 1st patient to be dosed with gene therapy happened in Canada under Taysha Gene Therapies clinical trial. The buzz and excitement was palpable during the conference. This was a momentous day for Rett syndrome community. The potential for a treatment that addresses the underlying cause of disease and slows progressions or potentially prevents the onset of disease with early intervention is truly remarkable.
Also, Nature Medicine Published Results from Pivotal Phase 3 LAVENDER™ Study Evaluating DAYBUE™ (trofinetide) Efficacy and Safety in Patients with Rett Syndrome. Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study | Nature Medicine
Hope is alive and well for the future of Rett syndrome. With the commitment and dedication of so many, one day we will have available precision medicine where families can choose what treatment will be best for their loved one. This reality is near and I for one am extremely excited about what’s to come.
Many thanks for this opportunity to attend this meaningful meeting.
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