Halloween Contest: Get Creative!


As summer draws to an end and Walgreens begins displaying fun-sized bars of snickers and tempting bags full of candy corn, we have to ask ourselves the serious questions. What will my child want to be for Halloween? Where are we going trick-or-treating? Are they still young enough that I can eat all their candy without them really noticing? But for those with children who have rare diseases, … [more here...]

Forbes Reports: Rare Disease Patient Voices Bring Change To The Clinical Trials Process

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by Stephen Smith The voices of rare disease patient advocates play a unique and critical role in the modernization of the clinical trials process, … [more here...]

A Visit With a Princess: Alana with XLH


Last night was rough. I had just gotten back to my parents house in Orlando from Clearwater, Florida after a successful hometown premiere for the … [more here...]

Stirred by the 2014 Patient Advocacy Summit to Talk About the High Cost of Treatment


I tuned into the RARE Patient Advocacy Summit via the free livestream webcast specifically to hear Dr. Jim Olson’s Innovations in Science Presentation … [more here...]

Why Are Genetic Conditions So Hard To Understand?


Although technically Rare Disease Day is in February, Rare Disease Day is actually every day for us. One of our family members has a rare genetic … [more here...]

Global Developmental Delays in Preston Leads to Birk-Barel Syndrome Diagnosis


Preston Kelley was born December 27, 2013. He spent the first three months of his life in the NICU at Children's Hospital of Orange County CA. He was … [more here...]

Bouncing for Shwachman-Diamond Syndrome (SDS)! Long Island Parents Hold Bounce-House Fundraiser

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Balancing fun while managing the seriousness of their son's rare disease is something the Del Re's have learned to do. Now 2 years old, Anthony was … [more here...]