July is National Cleft & Craniofacial Awareness & Prevention Month! Cleft and craniofacial conditions affect thousands of infants, children, teens and adults in the US each year. Some are born with…
Last month at the United Leukodystrophy Foundation’s Family Conference, special education teacher Sharon Bergfeld, MASE, LBSI, LBSII, led a break out session on Student with Disability/Schooling Advocacy. The session provided…
According to a story from PharmaTimes, the pulmonary arterial hypertension drug selexipag, developed by Actelion, was unable to make the cut to obtain coverage on the NHS in England. The…
Recently in Charcot‐Marie‐Tooth news, progress has been made in identifying better testing to use as outcome measures for people living with Charcot-Marie-Tooth disease type 1A. A study called “Outcome measures…
This is a continuation of An Interview with the Unstoppable Helena Baker. We had just learned about the multiple decades it took for her to receive her diagnosis of Fibula…
According to a story from PBS, the FDA has been approving new treatments at a noticeably faster rate than it has in the past. As a result, it appears that…
BuzzFeed News reported a few weeks ago about the tragic story of 16-year-old Sara Manitoski who died of Toxic Shock Syndrome: a rare condition with a very common source. Toxic shock syndrome…
Happy Friday, everyone! We hope everyone's drinking water, wearing sunscreen, and taking a little time to relax this summer. As we get ready for the weekend, we've gathered four recent…
According to a story from bioportfolio.com, two companies, Ionis Pharmaceuticals, Inc., and its affiliate Akcea Pharmaceuticals, recently announced that its product inotersen (to be marketed as TEGSEDI) has gained approval…
According to a story from NPR, plastic straw bans have been gaining traction across the U.S. One of the most shocking examples of planetary degradation as the result of human…
According to a story from the Deccan Chronicle, researchers may have discovered a new treatment for Dupuytren's contracture that could offer substantial benefits over currently available treatment options. The scientists…
Bruce Howell is 63 years old living with retinitis pigmentosa. He’s been legally blind since age 37. As a result, public transportation is a critical service to him. Without the…
According to a story from CNN, Nick Foles, who plays for the Philadelphia Eagles, helped lead the team to victory at the Super Bowl, and was awarded MVP for the…
If you look on Ed Sheeran's Instagram you will see a recent photo of him and a beautiful eleven-year-old fan. But this is not just any fan-- the picture holds…
Duncan Avery is father to two children, Kalea, and Noah. At age six, doctors discovered Kalea had a tumor in her brain. They would successfully remove it via surgical methods.…
Most of us are familiar with Parkinson's Disease. Parkinson’s disease is a central nervous system disorder affecting movement. It is a progressive disease and develops gradually, sometimes starting with a subtle tremor in…
Who needs a shot of feel good today?! Young Skye Savren-McCormick was diagnosed with juvenile myelomonocytic leukemia in 2016 and required a bone marrow transplant - and Hayden Hatfield Ryals…
Helena Baker is the Vice President of Clinical Strategy at Medical Research Network, a Nurse, and also, a rare disease patient. I had the opportunity to interview her for a…
According to a story from medicalxpress.com, early tests of a gene therapy for X-linked myotubular myopathy are showing promising results. This could represent a major breakthrough for this disease, which…
Cleveland Clinic’s first every three-way kidney transplant has taken place. It involved six people in total: three donors and three recipients. The full article can be read here, at the…
When you think of arthritis, naturally the first thing that comes to mind is an older person - and with good reason, since artists is more common in people over…
For any John Oliver fans out there, you know his thing is deep-diving into topics of importance - from beauty pageants to international elections to failing American infrastructure - just…
Happy Independence Day Weekend! As the firework shows wind down, we want to wrap up the week with three great contributor stories, and one research update. First, we have some…
Dewayne Johnson used to work as a school groundskeeper. Now, some days, he can’t manage to speak as a result of the cancer he lives with. Now, despite professional expectations…
July 13th is Gastrointestinal Stromal Tumors Awareness Day! And that "13" has extra relevance. 5,000 Americans are diagnosed with Gastrointestinal Stromal Tumors (GIST) each year. That’s 13 people diagnosed each day.…
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.
