According to a story from The Atlantic, the continual development of gene editing technologies like CRISPR have the potential to cure a substantial portion of rare diseases that are linked…
According to a story from Scoop Independent News, speech therapists from the Waikato District in New Zealand have been using telehealth technology to great effect in order to deliver speech…
According to a story from MarketWatch, the biopharmaceutical company MediciNova recently announced that the company's product ibudilast was granted Orphan Drug designation by the FDA. The designation was granted for…
If you think you have to be middle-aged or older to check your cholesterol, think again. Laura Livesey is a relatively young blogger (you can check out her blog, Confidence…
The clinical research process is a thorough and time consuming method in which new drugs, therapies, and medical devices are rigorously tested before being made available to the public. Through…
How it all started Joy Milne is a retired nurse who helped diagnose her husband with Parkinson's disease. 12 years before the official diagnosis, Joy noticed a change in her…
A past, current, and future problem – underrepresentation in clinical trials, particularly for rare diseases. Why does representation matter? Besides, you know, the basics of equality, drugs can react differently…
Topline results have been shared from a Phase 3 study of the investigational drug givosiran in patients with acute hepatic porphyria. For more detailed information you can view the source…
Many people express skepticism about the pharmaceutical industry. If not skepticism, than concern, if not wholesale distrust. For every few negative stories, however, there is at least one positive example.…
According to a story from the Bensalem Times, David and Catherine Woodley's adopted daughter Abby recently underwent a transplant operation that saved her life. David and Catherine adopted Abby three…
According to a story from yahoo.com, a six year old boy with from Texas named Mason Motz recently began speaking for the first time after a visit to the dentist's…
I have Gaucher disease. I often wonder what it would be like to not have this disease. But then I stop and redirect my thoughts. I’ve spent too much time in the…
According to a story from the Milton Keynes Citizen, a local boy named Josh from the Milton Keynes community with the rare lysosomal storage disease Niemann-Pick disease type C will…
Shriners Hospitals for Children® has announced their motion analysis centre consortium. The hospital’s Motion Analysis Centre is used to evaluate the movements and gait of children and teenagers. To read about…
Joe Marksteiner, a 22-year Air Force veteran and former gymnast, won a title at the 2013 World Powerlifting Championships at the age of 60. This year, now at 65, he…
According to a story from Medical Xpress, an international group of scientists from the US, France, and Spain may have discovered that an antifungal agent commonly used to treat skin…
According to a story from Greenville Online, the Greenville Health System has begun implementing a comprehensive treatment program for patients with sickle cell disease and its most severe and common…
According to a story from BioSpace, the biotechnology company BioElectron recently announced that the results from its Phase 2a clinical trial of its experimental product EPI-589 are looking positive. EPI-589…
Show of hands - who has ever indulged in a little beauty pampering? A mani/pedi? A haircut? A shave? Sure, that was a trick question - who hasn’t?! And that’s…
Just Searching for Answers When diagnosed with a rare disease, cancer, or other illness, it makes sense that you’d research every type of treatment on the market. However, there are…
According to a story from globenewswire.com, the pharmaceutical company Portola Pharmaceuticals recently announced that its investigational product cerdulatinib has earned Orphan Drug designation from the US Food and Drug Administration…
According to a report by medicalexpress.com, researchers recently discovered a new genetic condition. Though researchers previously described the condition in animals, no human evidence had yet been found. Michigan State…
According to a story from Business Wire, the biotechnology company Abivax recently released the results from its Phase 2a clinical trial. This trial was testing its investigational product ABX464 as…
Misha Walker and her husband Mike have spent the summer in England, meeting with families who have children with a rare condition called arthrogryposis multiplex congenita (AMC). In the…
According to a story from Research & Development, increasing collaboration between a variety of different organizations is helping to propel and improve the develop of new drugs for the treatment…
© Copyright 2024 Patient Worthy
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.
