Elizabeth Jane Allen, known best as "Betty", will be honored this week for the wonderful woman she was and the lasting impact she has made on the cystic fibrosis community, reports…
According to an announcement from ema.europa.eu, the European Medicines Agency (EMA) recently granted market authorization for a new treatment for alpha-mannosidosis. The new drug is an enzyme replacement therapy called…
Maddie Holt from Everett Washington is a 5-year-old with severe disabilities. She can barely hear or see, and she certainly can't walk or talk, reports Maine Public. Unfortunately for her,…
According to a story from CBS News, Hazel Hammersley was diagnosed with neuroblastoma at age two. As a result, she was forced to fight for her life at a very…
According to a story from Bronchiectasis News Today, the results of a new study suggests that bronchiectasis may trigger autoimmune inflammatory disease. The study found that the condition was frequently…
The Orphan Disease Center in association with the University of Pennsylvania and the Loulou Foundation just announced their 2018 Pilot Grant Program for CDKL5 Deficiency Disorder. CDKL5 deficiency disorder (CDD)…
Just this last June, a new drug successfully used to treat metastatic breast cancer patients in England, has been denied for a third time by Scottish Medicines Consortium (SMC), as…
The Alsabaa family not only has to deal with their 6-year-old son ,Tony, fighting children's idiopathic arthritis, but they are also not allowed to be in the States with him…
Shaquem Griffin, a linebacker from the University of Central Florida receives a ton of buzz because he's an amazing athlete. Point blank. As he should. Originally reported by Rare Disease…
While she's constantly smiling and full of energy, little girl Makenzie Cadmus struggles with a rare condition known as epidermolysis bullosa, reports News 12 Long Island. The rare disease has…
Just recently reported by BBC News, Jake Coates made a hard decision to stop attempting to have a child through the surrogate route after two failed attempts. Not even two…
University of Glasgow scientists might have just come across an incredible new approach to providing patients with pharmaceutical medications on demand, reported first by International Business Times. This new technology…
According to a story from Independent, cumbersome delays and possible funding cuts are severely hampering England's National Health Service (NHS). These delays are proving very costly for patients with long…
Zaida was 3-years-old attending her normal yearly check-up with expectation that all was well, yet they quickly learned six days later of her terrifying cancer diagnosis, reports Daily Camera. Little…
Tyler Chambers, 9-years-old, suffers from scoliosis, with a 65 degree bent spine. His mother is fighting to get him the surgery he needs to rid away his intensifying pain, reports…
According to an article from Newswise, a team of researchers are studying tissue engineering for the repair of knee injuries such as a torn meniscus. The meniscus is the section…
NBCNews Chief Foreign Correspondent, Richard Engel, opened up about one of the hardest phone calls he ever had to receive-- the call where he received the diagnosis of Rett syndrome for…
On January 30th, pop singer Jordin Sparks' step-sister Bryanna Jackson-Frias lost her battle with sickle cell anemia, reported People. Complications surfaced that sent her to the ICU. Jordin posted to…
Chloe Fernandez is an 11-year-old with primary ciliary dyskinesia, and that hasn't stopped her from being an advocate for the disease, reports Los Angeles Times. When given several amazing opportunities…
A new effort to raise awareness and funding for rare disease is underway. SMA News Today reported that NORD, the National Organization For Rare Disorders, organized this campaign to challenge…
According to a story from Cancer Updates, Research & Education, the Food and Drug Administration (FDA), recently approved the use of Trisenox injection in combination with tretinoin as a treatment…
Welcome back, Patient Worthians! This week, we have an opportunity for Parkinson's patients, and an update on an Amyloidosis app. We also have two stories of teenagers whose lives suddenly…
Emma and Tripp Mattingly share the same infectious personalities of energy and happiness, but they also share the same rare genetic disease known as LPIN1, reports The Lebanon Enterprise. Living…
There aren't many families like the Bradley's-- not just because they face the loss of their 10-year-old son who passed away recently due to brain cancer, but because of their…
Adam Murphy, a reporter for CBS46 learned of his mother's diagnosis of Progressive Supranuclear Palsy, PSP, in 2010 and decided to use his reporter platform to help her, originally reported…
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