IamA mom to a child with the ultra rare disease Hunter Syndrome trying to make a difference on World Rare Disease Day (today). AMA!
My short bio: I'm Melissa Hogan and I'm doing this AMA today on World Rare Disease Day on behalf of my 8yo son Case and his many friends fighting Hunter Syndrome (Mucopolysaccharidosis II), a disease that affects around 2,000 kids in the world. I administer my son's weekly infusion (a drug that costs around $9,000/week), advocate on his behalf, and enrolled him in a clinical trial 5 years ago. Some find it interesting that he got a medical tattoo when he was four years old as part of it.
I produced an awareness and fundraising campaign called Project Alive, an idea that came from a photo my son's kindergarten teacher sent home. We received support from celebrities like Jared Leto, Alexander Skarsgard, and Pete Carroll. For more information, visit ProjectAlive.org.
I also serve as a Patient Representative to the FDA, run our nonprofit foundation called Saving Case & Friends, and write and speak on rare disease issues. I can’t wait to answer your questions.
EDIT 6:30pm: Stepping away for a few hours to give my son his infusion. Real life of rare disease beckons. I'll pop back in later tonight and over the next few days and answer any additional questions. Thanks for all the great questions and kind comments! -M