When you hear your newborn's been diagnosed with a rare disease, you may feel like you have to become a scientist in order to raise them. Parenting a child without…
Were you into science at school? It’s funny, I never was, but I discovered a consuming interest in all facets of scientific discovery as an adult. Maybe I wasn’t encouraged…
Young adults with spinal muscular atrophy (SMA) have the same interests as those without SMA. You may want to go to college and pursue you dreams. Or you do you…
Want some more news, events and announcements on HAE? We got 'em! [one_half] [/one_half] [one_half_last] Get the Most Updated HAE Treatment Information Treatments Are Available: What Now? New,…
Paige Hildring is nine years old. She’s one of 108 people in the entire world with PURA Syndrome. PURA Syndrome is a neurodevelopmental disorder and those who suffer from it…
Short bowel syndrome, which is also called short gut, occurs as a result of someone having at least half of their small intestine removed, and sometimes the removal of a portion of…
For those of us who aren't familiar with Tardive dyskinesia, here's a little background: Tardive dyskinesia is actually something like a severe, often long-lasting side effect to drugs that treat…
Researchers have reported an early victory in treating sickle cell anemia with a new gene therapy. This could possibly lead to a cure. According to the U.S. National Heart, Lung, and…
The HCU Network of America is not only trying to spread awareness and encourage research for homocystinuria, they were founded to serve as a resource for those currently living with…
When it comes to the topic of severe fatty liver disease there is good news and bad news. In the spirit of leaving on a good note, let’s get to…
Pregnancy complications can arise no matter how healthy the expectant mother is or how careful she's been to protect her precious bundle of joy. Certain complications are minor—while others pose…
Want some more news, events and announcements on Narcolepsy? We got 'em! [one_half] [/one_half] [one_half_last] A Narcolepsy Miracle Drug? First Patient In Phase 3 Study Evaluating Treatment for…
Have you ever heard of “ex vivo” gene therapy? If you haven't, it means removing some cells, modifying them, and putting them back in. The goal? Curing the diseases that…
Want some more news, events and announcements on Gaucher disease? We got 'em! [one_half] [/one_half] [one_half_last] Promising Molecule Identified as Possible Treatment for Gaucher, Parkinson's Diseases NICE Changes Mind…
The Neurodegeneration with Brain Iron Accumulation (NBIA) Disorders Association was established in 1996 to serve and support all those affected by PKAN, PLAN, MPAN, BPAN, and CoPAN. Of these, PKAN or…
I think most people experience eye problems at one point or another. Maybe your child came home with "pink eye," and before long, everyone in the household had itchy, burning…
Want some more news, events and announcements on Dravet syndrome? We got 'em! [one_half] [/one_half] [one_half_last] Opko Health Inc. is Closing In on a Treatment for Dravet Syndrome…
Second grade. So big but so little. An academic year filled with spelling and vocabulary words, simple math fast facts, and learning how to use a keyboard. And maybe at…
Welcome to Friday Patient Worthians! This week we have a PW Contribution on one woman's battle with Stiff Person's Syndrome. We also have a piece on using fear to serve you in…
The below article was originally published on May 31st, 2017 on the International Pemphigus and Pemphigoid Foundation website, and is being republished here with permission. It wasn’t easy getting to…
What’s the biggest nightmare of any parent? Is it the fear of a kidnapper? Is it some horrible, lifelong medical condition that needs constant medicine or repeated hospitalization? Is it…
Be on the lookout for this amazing sickle cell anemia blog! Caregivers, friends, family, and patients of sickle cell, there is a new blog committed to raising awareness about sickle…
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…
It’s so easy to nitpick and find things wrong with organizations. It’s much harder to see what it is they're doing that's good. One particularly fun punching bag is the United…
Recently diagnosed with a rare disease and don’t know where to turn for more information? Have a loved one with a rare disease and struggling to understand the science behind…
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.
