The Chan Zuckerberg Initiative and Citizen Health have announced a strategic investment and partnership to drive drug development and accelerate cures for rare diseases. Ctnnb1 Connect & Cure is proud to be part of this rare disease movement, with many of our members already using Citizen Health to share their de-identified data and contribute to critical…
Researchers at Tufts University School of Medicine and the Graduate School of Biomedical Sciences (GSBS) have identified a small molecule that, in mouse and human cell models, rectifies the underlying molecular cause of a rare genetic developmental disorder linked with motor and intellectual disabilities and some types of autism spectrum disorder. Their most recent research, published in EMBO Molecular Medicine, provides the…
Dr. Michele Jacob at Tufts University and collaborators from Johns Hopkins are working on a novel mRNA approach to help our Dragonflies. This short animation describes this type of treatment. https://youtu.be/nkG1RvnZvPg?si=cecwsoSlenn-Up6v
Tonya is back to explain how a single drug compound could help our Dragonflies. https://youtu.be/-YBIjRnwEeo?si=SlBruZa5VbVL65Vu
CTNNB1, Funding Research, Advocacy, and promoting Awareness. Like, did you know that the number one genetic cause of Cerebral Palsy is CTNNB1 Syndrome? https://youtu.be/g05-YGNVexU?si=ehakhR83wpRTz_Ry