University of Louisville Grad Fulfills Lifelong Dream Despite Living with MPS1

On Saturday, 29-year-old Ryan Dant graduated from…

Rare Disease Report: China’s Rare Disease Policy (or Lack Thereof)
By James Radke of Rare Disease Report China is about to publish its first list of national rare diseases.   Given the population of China… Continue Reading
Learning to Be The Best Patient I Could Be: Growing Up with Homocystinuria
By Danae Bartke I was diagnosed with Homocystinuria in 1995 at the age of 10. We discovered the diagnoses because my younger brother had… Continue Reading
FDA Grants Priority Review Status to Ultragenyx
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and… Continue Reading
Texas Teen with Rare Disease Gets Wish Granted
Make-A-Wish Foundation volunteers Thursday surprised a 13-year-old Central Texas girl who has a rare disease with news that her wish to go… Continue Reading
Shire’s Swelling Disorder Drug Moves Closer to U.S. Approval
By Natalie Grover (SOURCE) Successful late-stage data on Shire Plc’s, long-acting preventative treatment for hereditary angioedema (HAE)… Continue Reading
Global Genes Rare Disease Patient Impact Grant Goes Towards The LHON Genealogy Project
As a 11778G>A mutation carrier, Lissa Poincenot, founder of LHON.org, is forever connected to the Leber hereditary optic neuropathy (LHON)… Continue Reading
NFL Player Struggles with Complex Regional Pain Syndrome (CRPS)
(SOURCE) After just two days around the Seahawks, Tammy Jones is all in. “I love the Seahawks family already. I feel like I’m leaving… Continue Reading
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