Happy New Years! We're back with a batch of new article! This week, we're highlighting four stories: an interview on 20 years with dystonia, a young woman stuck in a…
As originally reported in EurekAlert, creating effective rare disease pharmaceuticals is difficult. Yes, because it is expensive, there’s a small population to use in trials, and there’s limited research. Sometimes…
The human spirit can be a remarkable force. Robb Freed cycled thousands of miles by himself to raise awareness for epidermolysis bullosa (EB) -- a rare illness that took the…
The annual Code Talker award was presented during an engaging ceremony at the National Society of Genetic Counselors’ annual conference. Every year, patients submit essays nominating their genetic counselors for…
Treatment for ulcerative colitis differs based on the individual. Where the inflammation occurs, the severity, and the preference of the patient can all influence the treatment. For those with moderate…
As originally reported by the Oklahoma Medical Research Foundation; Tonja Martin was running through her retirement savings for her medical care. She spent $3,400 every month to cover treatment for…
TrialJectory is an organization working to match bladder, breast, colorectal, melanoma, myelodysplastic syndromes (MDS), and other cancer patients with the clinical trial that is right for them. They have successfully raised…
Who is most affected when we talk about creating solutions for rare diseases? Those who live with them and their families. Patient communities know what they need and feel the…
Savara Pharmaceuticals has recently interviewed Dr. Tisha Wang, who is a pulmonologist and critical care physician at UCLA. Dr. Wang is also an advocate for autoimmune pulmonary alveolar proteinosis…
This is NSGC 2019 Conference Highlights Part 2, click here for part 1. An early morning session on the final day of the conference featured a personal story shared by…
Written by: Paul Pavao The most important thing I can tell you is that Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) can be survived. As of 2019 survival requires a bone…
Regeneron Pharmaceuticals has recently announced the results of Phase 2 of their study of pozelimab, which is an experimental treatment for paroxysmal nocturnal hemoglobinuria. Current therapies for this condition…
Genetic Counselors are an incredible resource – they can help assess your risk of developing a condition, the risk of your children inheriting a condition, and what potential treatment or…
“I feel like I’m in a huge dream that nobody can get into,” Noemie explained, describing her weeks of intensive sleep during which she slept 20 hours a day, the…
Sometimes in moments of tragedy, there is a bright silver lining that helps us learn, and that's exactly what Amber Briggle was aiming to do. Back in October, Amber posted…
No healthcare professional wants to hear that a drug being studied in a clinical trial did not meet the endpoints. It means that after countless hours of hard work,…
It is estimated that about 10% of people in the United States have rare diseases. But what makes a disease rare? How does this distinction affect diagnosis and treatment?…
As originally reported in MSU Today; in new experiments on mice, researchers looked into how the TS gene is related to autism. Tuberous sclerosis is a rare disorder known for…
As originally reported in the Hindu Times; getting healthcare for a rare disease is hard, in general. 95% of rare disease patients have no therapy options available. Thats 380 million…
In the world of rare diseases, progress can often feel like a slow and agonizing slog. In many cases, this is the unfortunate reality. However, a lot can change in…
As originally reported in Pulmonary Hypertension News, sleep is a natural cleanser for the body. During the nights, our brain imbeds our memories, removes metabolic waste, and heals itself and…
As originally reported in the Cambridge Independent; rare disease treatment is full of potential. In part, because there aren’t many. This is not for lack of ability, but for lack…
As originally reported in Medscape; many rare disease patients know the frustration of not having any drugs or treatments available for their disease. 95% of rare disease patients have no…
If you are unable to work because of a medical condition, you may qualify for disability benefits from the Social Security Administration (SSA). The SSA oversees two different disability programs, Social Security Disability…
According to a story from BioPortfolio, the drug companies bluebird bio, Inc. and the Bristol-Myers Squibb Company have recently announced positive final results for their phase 2 clinical trial. This…
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