Sangamo Therapeutics shared some big news about the search for Mucopolysaccharidosis II (MPS II) treatment. They reported that they have, for the very first time, used experimental in vivo gene therapy to treat…
Sean Kisielnicki's life is ruled by Hunter syndrome. This rare condition not only affects him, but takes a toll on the entire family. The morning routine is always the same.…
Karl Swaine is nine years old. He has a rare progressive and genetic disorder called Hunter syndrome. This disorder prevents the body from breaking sugar molecules down. Throughout the world,…
Each year on the very last day of February, Jeff Leider makes a detour on his Hunter syndrome (mucopolysaccharide II) journey to travel from his home state of New Jersey to Washington…
Okay, I’m going to be perfectly honest: I don’t quite know what to make of this. On the one hand, you have some excellent news for people living with mucopolysaccharidosis…
One teenager with Hunter syndrome doesn't let anything hold him back. This doesn't go unnoticed. Hunter syndrome is a rare inherited disorder which prevents production of a key enzyme that…
Everyone has a story. Every person has something that they can share. Reading about another person’s story can help you and your family cope with Hunter syndrome. Also, learning about…
To walk in the shoes of others is sometimes what it takes to truly understand their journey. So when I came across the blog, Jason and Justin's Journey x2 - Don't Stop…
Some lucky people live their whole lives only taking medication intermittently, like pain killers when they have a headache or acute pain. Maybe they take an anti-inflammatory when they have…
As the saying goes, “Many hands make light work.” Know what’s even sweeter? When those hands are little—little tiny hands that finger paint, swing on the playground, and play monster…
Every week, my son’s principal sends a voicemail to parents highlighting the week’s news and activities. At the end of the message, she signs off with a reminder to “Be…
Hunter Syndrome, is a heartbreaking twist of genetic fate, and we celebrate that the FDA has granted Rare Pediatric Disease Designation to REGENXBIO's RGX-121 Gene Therapy. Hunter Syndrome causes seemingly…
What are parents’ hopes for their children? Typically a happy, successful and long life. Children are often asked what they want to be when they grow up... but the growing…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
