Mark Cohen is a 28 year old lawyer who has a rare eye disease called choroideremia. Its genetic, and his grandpa, brother, and cousin were all diagnosed. He didn't receive…
A recent study conducted by a government agency, NIAID, offered preliminary evidence of Remdesivir’s benefit to COVID-19 patients. The drug is heralded as the first antiviral drug to improve COVID-19…
Maddie Nordhoy has a smile that can light up the room. She constantly brings joy to those around her, especially her family. In fact, she is even the inspiration for…
Social distancing sucks for everyone. Even introverts (like myself) are becoming antsy. Similarly, the coronavirus is scary for everyone, even those less likely to be effected by serious illness. However,…
By Jodee Redmond from In The Cloud Copy At one time, hearing a cystic fibrosis (CF) diagnosis was tantamount to a death sentence for a young person. The median survival…
When the 31-year-old woman showed up to Massachusetts General Hospital with a nasty respiratory infection, she was somewhat used to it. In fact, she had been plagued with respiratory problems…
Because I will be looking for a job from a rare but certainly not unique place of being, I decided to reach out to a mentor who lives with the…
U.S. company, Vertex Pharmaceuticals, is the creator of three treatments for cystic fibrosis: Orkambi, Symdeco, und Kalydeco. These drugs were unavailable in Switzerland, as Vertex and Swiss health officials were…
Recently, says Medical XPress, an international research team discovered that the drug therapy 2-deoxy D-glucose can slow cell growth. In patients with ovarian cancer, the drug can halt the growth…
According to a story from the CureDuchenne Blog, CureDuchenne founder and CEO Debra Miller announced that the organization has recently entered into a new partnership with the biotechnology company Myosana…
The Connect MPS Patient Registry was created in 2016 to learn more about Sanfilippo syndrome and mucolipidoses (ML). 24 advocacy organizations are a part of this registry, all of which…
HAE is not my story. But it’s definitely part of my story. Looking back, I realize that my late childhood and early teen years held some occasional, unexplained swelling events that…
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.
