Welcome back to Editor's Choice! This week we have an interesting take on Parkinson's treatment. We also have an empowering article from one of PW's rare disease contributors. We are all feeling the…
If dance is the universal language, then what does NC State’s annual Dance Marathon have to say--especially to type 1 tyrosinemia (TYR)? A lot, it turns out. The numbers seem…
After lobbying during Rare Disease Week on Capitol Hill a couple of weeks ago, I was convinced we were pretty successful with our congresspersons in showing them how vital NIH…
I recently covered an author who had some solid advice for drug makers in regards to the rare disease pricing problem. The following drug maker may or may not have…
Ladies and gentlemen, it's that time of year again, when I put on my superhero cape, I ascend to the top of the highest mountain, and at the top of…
The liver is a pretty important organ. Some people think of it as the place where alcohol goes. Your neighbor’s kid in the 6th grade will tell you that it…
It's really important to be able to recognize the signs Parkinson's disease in women. Most people identify Parkinson’s as an “old man’s illness;” yet thousands of women are diagnosed with Parkinson’s…
Canadian, Barb Charboneau, felt something was wrong with her around the age of 11. Years later, she was diagnosed with Tourette syndrome. Then it took many more years for her…
“Breaking The Sickle” is the name of a new children’s book. At first glance, you might think it is about destroying communist regimes, it is in fact a book about…
Friedreich's ataxia (FA) is a rare and painful disease. With few treatments available to treat the genetic mutation, most therapy is aimed at controlling symptoms. Clearly, these short-term band-aids aren't…
Treading water is difficult. Sometimes, that’s how it feels navigating a new diagnosis. It’s like trying to keep your head above water in a vast, foreign ocean, as waves of information…
Chucky Bartolo of Lovin' Malta introduced Jake Vella to the world yesterday. Jake suffers from the rare disease ROHHAD. While he was a regular kid for the first five years of his…
The key to solving ANY problem is knowledge. And so the same goes for raising awareness for those living with any type of illness. The more we know about the…
Sometimes, the race to develop breakthrough treatments for rare and devastating illnesses can feel less like the Indy 500 and more like a game of Curling played by armless sloths.…
We are huge proponents of the 21st Century Cures Act, as it does so much for the rare community. However, there has been an unintended consequence of it's 2016 passing…
In the post-Sputnik era, science was king—or so says Peter Agre as he recalls the golden age of science. But, when is it wise to dismiss the “ivory tower” mentality…
Cure Sickle Cell is a nonprofit organization. One of their goals is to raise money for research. Sickle cell is an inherited disease. Moreover, the disease prevents red blood cells…
I was alarmed and yet inspired by a recent article I read. Narcolepsy affects nearly 200,000 people in the United States, but sadly goes undiagnosed and undertreated. Don’t let this…
Alicia Goss is a college student, and for a long time she didn't know she had a rare condition called Wilson disease. She just wanted to go out and have…
When Megan Howard looked down at her completed drawing, colorful lines intersecting to form odd shapes, she saw elements of “The Scream.” She thought it a fitting depiction of her…
I’ve found that people feel very differently about support groups for their diseases, including narcolepsy. Some seek them out. Others avoid them at all costs. Some might not like the…
One tie, two ties, red tie, blue tie... The list goes on and on for Doug Robins, a young Australian man who’s raising awareness for Duchenne muscular dystrophy (DMD) by…
Una de las (muchas) cosas que hace que la fibrosis quística (FQ) sea tan difícil de tratar es que hay muchas mutaciones de la enfermedad, algunas afectando a subconjuntos muy…
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…
It’s a scene no one anticipates. The doctor walks into the room where you, and maybe a loved one, are sitting. Clipboard in hand, the doctor pulls up a chair,…
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