I recently read a report on the inspirational Lakiea Bailey, who has a rare disorder called sickle cell anemia. This disorder targets her red blood cells. Sickle cell diseases can…
Could crizanlizumab be the next treatment to prevent pain in those with sickle cell disease (SCD)? Is this the medicine you've been hoping for? Sickle cell disease (SCD) is an…
Has your healthcare provider ever talked to you about using an FDA-approved drug for an unapproved use (sometimes called an “off-label” use) to treat your disease or medical condition? It…
Oh the joys of a 17-month-old toddler… I remember some of the typical behaviors of those days—the throwing heaving of everything and anything, the opening of the fridge a million…
Losing a child is a loss like no other—the most devastating experience a parent can face. And for one daddy who lost his little girl to mucopolysaccharidosis, a very rare genetic…
What are the reasons couples break up? When our relationships fail, we sometimes wonder if there was something we could have done to save them—or if there were warning signs we…
The U.S. Food and Drug Administration (FDA) approved Biogen’s SPINRAZA™ (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is the first and only…
According to an awesome story in DelRay Newspaper, Kelsey Diamantis had always dreamed of going to the Kentucky Derby at Churchill Downs to watch the horse race. While thinking about…
Imagine your father has just suffered a heart attack and must undergo open-heart surgery in order to repair the damage... Picture your baby niece or nephew was born with a…
You're happily tucking your toddler in for her afternoon nap when she starts to panic. Where's her favorite blankie and stuffed penguin? A peaceful nap won't occur without it! With…
Passion. Passion doesn’t persist. It is there, and then it is gone. Standing on the diving board is pre-passion. Falling toward the water is post-passion. It's the jump that's ripe with…
Everybody wants to win. Winning gives a certain pride and joy that keeps us wanting more. Winning becomes a motivating factor for a person to do her best so she…
Attitude is one of the most important elements of bringing together “who you are” with “what you do.” Attitude may not be everything, but it’s important enough that nothing else…
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…
Jim and Kristen Farrell have two children, Braeden and Kernan, both of whom have a progressive muscle-wasting disorder called spinal muscular atrophy (SMA). The disorder can take away a child’s…
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…
Regardless of whether labor is long or short, whether it is hard or easy, whether a baby is born vaginally or by cesarean, most parents recall the first hours and…
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…
Look closely. A long shadow may be clouding your future. It's the shadow cast by the pain and suffering in your past—the parent who wasn't there, the boss who humiliated…
One of my biggest struggles used to be living without a diagnosis and trying to figure out what to say to people. What was I supposed to say when I…
Our children bring us so much laughter and joy. Spending lots of quality time with our kids is proven to be good therapy; they benefit from wholesome interaction with their…
June-September is wedding season. One of the most frequented topics of conversation among friends is weddings. It seems I'm at a stage when no matter who I'm with or what…
A firefighter, also known as a fireman, is a highly skilled man or woman who works to combat and extinguish fires. They also take steps to prevent fires, act as…
With fishing and summer recreation seasons just around the corner, you could be exposing yourself to Lyme disease and not even know it. You might become just like one mother…
A little pain usually is a good thing. It's our alarm system. It's our body's way of saying, "Hey, that's hot... Get away before it hurts you!" However, when the…
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.
