
The Rosamund Stone Zander Translational Neuroscience Center strives to accelerate the development of therapeutic options for children with brain disorders by fostering timely and efficient translational research.
Our Advocacy and Research Partners
Featured News & Events
Base editing of trinucleotide repeats that cause Huntington’s disease and Friedreich’s ataxia reduces somatic repeat expansions in patient cells and in mice
RSZ TNC and Broad institute researchers utilize base editing to reduce the repetitiveness of trinucleotide repeats that cause Huntington’s Disease and Friedeich’s Ataxia.
Development of a Patient and Caregiver-Centered Pediatric Disease Concept Model for Kleefstra Syndrome
In collaboration with RSZ TNC researchers and support from IDefine – The Kleefstra Syndrome Foundation, a graduate student from the Boston University Genetic Counseling Program, Kristen Connors, developed a disease concept model for Kleefstra syndrome (KS). This patient and caregiver-centered research identified unique aspects of the condition that most greatly impact the patient population and their families. This data may be used to generate educational materials for families and clinicians, and direct the eventual development of KS-specific interventional treatments that target features that are most important to the KS community.
September 3-4, 2025: Global Genes Rare Drug Development Symposium (RDDS) in Boston, MA
September 3-4, 2025 in Boston, MA, at the Joseph B. Martin Conference Center at Harvard Medical School. Connect with leading experts and drive meaningful change in rare disease research.