The Rare Disease Research Hub (RDRH) will be an online platform where researchers, patients and families, and others interested in Alagille Syndrome (ALGS) are able to connect and collaborate with one another, and, through the synergy of information and data sharing, catalyze advances in rare disease science and discovery. Initially RDRH will be a pilot project focused on ALGS, available as a subscription service to researchers but free to patients/families who choose to participate. In the future, once proven successful, the platform will be expanded to include other rare diseases, perhaps starting with those, like Alagille Syndrome, that are in the Jagged1/2 and Notch 1/2/3/4 pathways, then branching to other rare as well as more common diseases.

RDRH is critically needed in rare disease research. It will be a place where investigators, who frequently have never met a person challenged by the disease that is the focus of their work, will be able to interact with those experiencing that disease to ask questions and broaden their understanding. RDRH will offer researchers the opportunity to discuss their ideas and collaborate with others working in the same or a closely related field, thus sparking new research questions and paths of inquiry. It also will enable those desiring to learn more about research as well as contribute to the growing body of knowledge about rare disease to participate more fully in the science and discovery.


“Mobilizing resources, facilitating connections, promoting unity, and advocating for a cure to inspire, empower, and enrich the lives of people affected by Alagille Syndrome.”