2023 Research Projects That Need Our Help Raising Funds For:
GOOD NEWS!!!!
October 2023
It was announced this month that there is the first of its kind, potentially transformative therapy for MECP2 Duplication Syndrome! It is called ION440. Treatment will begin sometime in 2024.
Thank you to CURE MDS: The 401 Project, Rett Syndrome Research Trust, and to all my friends and family who have donated to this cause. Your donations are making a difference!
In addition to ION440, there are two additional therapies to bring to the clinic: SiRNA and CRISPR.
October 2023
It was announced this month that there is the first of its kind, potentially transformative therapy for MECP2 Duplication Syndrome! It is called ION440. Treatment will begin sometime in 2024.
Thank you to CURE MDS: The 401 Project, Rett Syndrome Research Trust, and to all my friends and family who have donated to this cause. Your donations are making a difference!
In addition to ION440, there are two additional therapies to bring to the clinic: SiRNA and CRISPR.
Updates to Current Research
In late 2010, we joined efforts with the Rett Syndrome Research Trust (RSRT) in a collaboration to find a treatment for these similar diseases. RSRT is a non-profit organization with a tax ID number. Monica at RSRT was very instrumental in establishing the MECP2 Duplication Syndrome Fund. The Fund exclusively supports projects devoted to the study and means of treatment of MECP2 Duplication Syndrome. 100% of every dollar contributed is invested in research – not a single penny goes to overhead. To date, almost $5 million has been raised for duplication projects thanks to the combined efforts of many MECP2 Duplication Syndrome families.
Important Note: When making a donation to RSRT, please make sure you clarify that the money is for “duplication” research. To read more about our coloration with RSRT, please clink on the following link: |
The Researchers
Huda Y. Zoghbi, M.D.
Howard Hughes Medical Institute Baylor College of Medicine One Baylor Plaza T807 Houston, Texas 77030, U.S.A. (713) 798-6558 Fax: (713) 798-8728 E-mail: [email protected] 
Hilde Van Esch, M.D, PhD
Centre for Human Genetics University Hospital Gasthuisberg Leuven, Belgium E-mail: [email protected] 
SakkuBai Naidu, M.D.
Director, Neurogenetics Unit Kennedy Krieger Institute 707 North Broadway Baltimore, MD 21205, U.S.A. (443) 923-2778 Fax: (443) 923-2779 
Sarika U. Peters, Ph.D.
Assistant Professor Departments of Pediatrics and Psychiatry Vanderbilt University, Vanderbilt Kennedy Center for Research on Human Development (615) 322-9410 Email: [email protected] |