Helpful Links

Ethan & Nathan

Our Hospital

UF Health Shands Children's Hospital

Ranked in the top-50 pediatric hospitals in the country in a variety of specialties by US News & World Report, University of Florida physicians at UF Health Shands Children's Hospital provide the full spectrum of pediatric specialty services.

get to know your study Sponsor

Clinical Research Sponsors

Meet the companies that have made a commitment to helping those affected by the rare diseases and are currently funding our clinical trials.

Connecting patients and families

Patient Organizations and Foundations

These groups—many founded by parents within the rare disease community—help connect patients and families to education, research, and support services. Here are just a few of the organizations that work to raise both awareness of rare diseases and funds directed toward research while supporting their communities.

Other Resources you may need

Additional Caregiver Resources

This page is intended as a resource to gather relevant links and information in one place that have not been mentioned elsewhere.

Click. listen. feel.

Rare Disease Podcasts

You are guaranteed an emotional rollercoaster and practical thoughts that apply to many areas of life with these podcasts.

Our Preclinical Team

Powell Gene Therapy Center

The Powell Gene Therapy Center (PGTC) at the University of Florida has been instrumental in the development of newer, safer agents for the delivery of therapeutic genes to patients with genetic diseases.