Gene Editing What is gene editing? It's been a hot topic over the last few years, but basically the concept is this: instead of disabling a malfunctioning gene, researchers are…
According to a story from Pharmaphorum, there are a number of new treatments that have been introduced or have seen major strides in development over the last year. More patient…
According to a report by CheckOrphan, a recent study investigated the use of genome editing directly in the body. The research involved the use of these editing techniques in cases…
According to a story from the Santa Cruz Sentinel, the earliest results from a critical gene editing study could offer some encouragement for those hoping to treat rare genetic diseases…
According to a story from philly.com, four year old Ethan Lieber of the Roxborough neighborhood in Philadelphia is one of just 500 people in the US with Hunter syndrome. This…
According to a story from The Tennessean, mother Melissa Hogan has been inspired by her son Case's Hunter syndrome to start a nonprofit organization called Project Alive. The primary purpose…
Creative parenting. I find such inspiration when I hear about parents digging deep to adapt surroundings (or life) so their kids can deal with it better. A WBTV social media…
Easton was born at thirty-six weeks gestation on January 6, 2017. He was almost immediately taken to the NICU for something called PPHN (Persistent Pulmomary Hypertension), Easton was intubated for…
Today, Professor Elsa Shapiro and Dr. Christina Lampe presented "Cognitive Impairment in Patients with MPS II: From Disease Burden to Cognitive Testing" at Satellite Symposium, an event sponsored by Shire…
Gene therapy is on the rise, and it just might be exactly what many children suffering from genetic disorders need to save their lives. Today reported that an amazing organization…
REGENXBIO announced last month that the Investigational New Drug application (IND) is active for a Phase I/II clinical trial of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II)…
REGENXBIO Inc., a pharamaceutical company specializing in gene therapy, just shared that it launched a Investigational New Drug application to conduct Phase I/II trials on the new therapy they're developing,…
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…
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