Hereditary angioedema (HAE) has long been misunderstood. Many patients go for years without a proper diagnosis because the medical community knows very little about this rare condition. Part of my job…
Being married to the love of your life would mean that you know everything there is to know about one another. Physically, emotionally, and mentally. Right? Well, Gene is married…
I want you to think about spinal muscular atrophy (SMA), a genetic disease affecting the part of the nervous system that controls voluntary muscle movement. Then, think about the word: DIGNITY. Dignity…
The drug, Ceriliponase alfa from BioMarin, helped in a condition that has led inexorably to disability and death: Batten Disease – specifically CLN2. Families of kids facing this prognosis are…
Happy Friday Patient Worthians! Did you know it was World PI Week this week? We have a post on the role pets have with PI. We also have a story on a…
A special session on CGD will be presented at the 2017 IDF National conference. CGD is a primary immunodeficieny disease and is characterized by skin infections, both fungal and bacterial,…
If you or your loved one is living with idiopathic pulmonary fibrosis (IPF), scientists have proven that there is a link between exercise and longevity—especially for people who have IPF.…
A rare disease that is not so rare: Hormonal causes of high blood pressure have been considered rare but “can be as high as 50% among children and 30% among…
I was at a gala hosted by my local library about two weeks ago. It was a posh affair, as library galas go, and everyone was all dressed up. The…
Remember when PW reported on the Newborn Screening and Gene therapy for the Immune Deficiency SCID? Well guess what our friends at the Immune Deficiency Foundation are doing? The IDF…
Siblings! We all have our opinions. Not everyone has that close unified bond with his or her brothers and/or sisters, but one thing is certain: If the connection is there,…
When you’re a parent, the childhood milestones are many. Flash, blink, bam—and then the rite of passage to adulthood starts. Yeah, I know… I flew through those first 18 years…
Anger. Who doesn’t get angry? From big to small stressors in life, there are far far far too many triggers to choose from that can set us off. But, what…
The transition from childhood to adulthood—the “coming of age” of boys who become young men and girls who become young women—is a significant stepping stone in everyone’s life. However, the…
What would you do if you had a disease so rare that you went weeks or months without a diagnosis? What would you do if after your diagnosis, you told…
I’m barking mad! You know the old saying, “Let sleeping dogs lie,” right? Well, I couldn’t help but think of the 200,000 Americans living and struggling with narcolepsy because they’re…
A health crisis—just like epidermolysis bullosa (EB)—is different for everyone. But, most have one thing in common: Bad news blindsides us. We take a hit—a hard one at that. When…
Have you ever been lonely in a crowd? Have you ever been perfectly content all alone? Me too. And although it’s a little crazy to admit, I’ve also suffered from…
Not many people can say they’ve dedicated their life to curing the disease that could lead to their death, but David Fajgenbaum certainly can. In a fascinating Science Magazine article, David…
When I was in college, I went to the home of my biology lab partner to work on a project. While we were sitting at the kitchen table, I got…
While the national conversation on healthcare has recently and rightly focused on the political debate over the Affordable Care Act’s future, it’s worth remembering what’s happening on the “street level”…
For most of us, a nice stroll in the woods is a leisurely activity for relaxing or light cardio. But for disease ecologist Richard Ostfeld, a walk in the New York…
Primary immunodeficiency diseases (PI) weaken the body's immune system, leaving patients prone to infections. In very mild cases, the person may not even know they have PI, but in other…
Imagine watching movies with friends, not knowing that you have narcolepsy and struggling to stay awake. Your eyes keep rolling and you keep fighting to stay awake. Yet you cannot.…
BIG props goes out to a 43-year-old woman who’s living with sickle cell anemia! Having been accused of “jonesing” for more pain medication in the hospital, she quickly became sick…
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.
