Advocacy efforts play an essential role in building stronger systems of care for rare diseases. By elevating the experiences of families and engaging policymakers, organizations such as the Ivan & Joan Foundation are helping move rare disease awareness and care forward in their communities.
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Jakub Tolar
Dept. of Pediatric Hematology-Oncology | MD, PhD | Minneapolis, MN
Dr. Tolar’s research focuses on finding new ways of treating children with lethal diseases – cancer, inborn errors of metabolism, and devastating genetic disorders – using stem cell transplantation. He is also looking for safer and more effective methods of repairing and using a patient’s own cells in diseases such as epidermolysis bullosa, mucopolysaccharidosis type I (Hurler syndrome), Fanconi anemia, and dyskeratosis congenita.
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My name is Kambri, and I’m 17 years old. I was diagnosed with Fanconi anemia when I was five. Before my diagnosis, I just didn’t feel good for a while. I had frequent nosebleeds, bruised easily, and was tired a...
What I hope for most is a cure. And until then, I hope for Paige and others with FA to live lives as close to normal as possible, filled with opportunity, independence, and happiness.