August 21st is the online registration deadline for this rare disease event! The site says the 15th and the 21st, so either way, you'll want to check out the details…
A North Carolina-based research group has just developed research that indicates a potential stem cell treatment for lung fibrosis conditions such as cystic fibrosis (CF) and idiopathic pulmonary fibrosis (IPF).…
All you have to do is look around. If you do, you’ll see the rate of progress accelerating. Look at anything modern. Some people liken this to Moore’s Law concerning…
When I was in my 20s and 30s, I performed in a lot of musical theater performances— “Music Man,” “Big River,” “Carousel,” “You’re a Good Man, Charlie Brown.” I was…
Right up front, I'll admit I’ve done this: You’re in a parking lot looking for a parking space when you pass a handicapped spot. You see the placard that gives…
According to the AKU Society, Flavia was only a few weeks old when her parents noticed that her urine had stained her diaper an unnatural brown color. Flavia and her…
Inspiration is everywhere—from the words of your favorite writer to the blooms and leaves on your morning walk. You just need to open your eyes, and breathe it in. Sometimes, inspiration just…
Sancilio recently completed enrolling children with sickle cell anemia/disease in a Phase 2 clinical trial to evaluate a new therapy called Altemia (docosahexaenoic acid). The SCOT trial, as it will…
Global Genes is hosting its annual Tribute to Champions of Hope event for the rare disease community. Here's all you need to know about it: What: A blue-tie cocktail hour and dinner event…
Hema Biologics have recently released something even cooler and more inventive than just their traditional hemophilia treatments. They are way on top of their game and know exactly how to…
Your hear that? It's the sound of the future knocking on the door! The MIT Technology Review reports about the first-known experiment to edit human embryos in the United States using a…
What should you know about Tourette syndrome? How can you explain it to your child? These are probably just a few questions you may have if your child has been…
Happy Friday! This week, we have a story that might break your heart about a woman who battled cystic fibrosis. But we also have a story that might inspire you about a…
Trigeminal neuralgia (TN), or extreme, sudden facial pain associated with the 5th cranial nerve, occurs frequently in patients who have received a multiple sclerosis (MS) diagnosis. In a survey with 8,590 respondents,…
Much like the United States’ National Organization for Rare Disorders (NORD), E-Rare in the European Union was launched over a decade ago to bring together organizations that fund research for…
According to Dr. Steven Carson, of all the autoimmune disorders that are both chronic and joint-related, Sjogren's syndrome remains the hardest to manage. Standards of care designed to guide doctors…
The United States FDA just granted Orphan Drug Designation to Napo Pharmaceuticals for Mytesi (crofelemer) in the treatment of short bowel syndrome (SBS). SBS is a rare disorder in which…
Orphan drugs, for those of you who don’t know, are those designated especially for rare diseases. They respond to a public health need, but not necessarily an economic need by…
In recent news in the oncology community, the FDA has approved orphan drug designation to a new drug, SurVaxM, for glioblastoma. Glioblastoma is a rare type of brain cancer that…
Sa-Nakht was an ancient Egyptian pharaoh who lived circa 2700 B.C. While there are many tall tales surrounding his reign, one that is especially tall, literally, and not figuratively, is…
If you remember reading our story on 12-year-old Markie DeVoe and his choroideremia, we have an important update for you. If you haven't read the story yet, you can find…
A new study pinpoints faulty genes as another potential cause behind hemiplegic cerebral palsy. Until now, there was an insufficient amount of information to verify that genetics was part of…
Theravance Biopharma recently proudly announced the results of a successful Phase 2b trial for their gastroparesis treatment, Velusetrag. The results showed that this treatment safely and effectively improved symptoms and normalized…
Most things have multiple functions. The tire iron you use to jack up the car when changing a flat tire? It can be used as a self-defense weapon if an…
If you attend a conference of rare disease families, you might not even notice that many of those who are helping out with all aspects of making sure that everything…
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.
