• Research for Therapies

    Basic laboratory research in rare diseases leads to understanding the molecular mechanisms that cause disease. Researchers apply the knowledge to identify biological targets or biomarkers that are used to determine effectiveness of therapeutics. Advancing technology and breakthroughs in genomics and biochemistry have accelerated the time to clinical trials and drug development.

  • Supporting Patients

    Along with our research, the center’s mission is to support rare disease patients in their quest for knowledge, resources, support and treatments. To further expand our reach, faculty and staff, along with Notre Dame Students, engage in coursework, clinical research, community outreach and patient engagement.

  • Join us in our Fight

    Donations support faculty, fellows and students to carry out basic, translational and clinical research to develop new drugs and additional therapies for rare and neglected diseases, in partnership with patients. We are humbled by your trust and faith in Notre Dame to transform a child's life and are always grateful for your donation.