The Isaac Foundation The Isaac Foundation was established by a man named Andrew McFadyen. He created the organization in 2007 in honor of his son who is diagnosed with the rare…
A new study conducted by researchers in Japan has uncovered a new potential way to diagnose Fabry disease. Fabry Disease Fabry disease is an illness which inhibits the patient from producing the…
Glioblastoma Glioblastoma is a rare type of brain cancer. Sadly, it is the most aggressive form. The median life expectancy for the disease is between 11 and 15 months and…
Sumaira Foundation Sumaira Ahmed established the Sumaira Foundation so that fewer rare disease patients would have to face the experience by themselves. Sumaira Ahmed is diagnosed with neuromyelitis optica spectrum disorder…
Here's a shocking fact for you- individuals don't always conform to averages. Despite this, we use averages as an analysis for so many things. As it documents the majority, its…
Critical Path Institute The Critical Path Institute, or C-Path is a nonprofit organization which was established in 2005. It has a US branch, headquartered in Tucson, Arizona as well as…
Epidermolysis Bullosa Epidermolysis bullosa (EB) is a rare condition affecting approximately 500,000 individuals globally. Patients in the United States and Europe account for 50,000 of these individuals. The hereditary condition is…
At the heart of rare disease treatments is research. Currently, 95% of all rare diseases still have no approved therapies. Although there has been an increased focus on rare diseases…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is actually a term used to classify a few different conditions. Two of these are Crohn's disease and ulcerative colitis. These are rare…
Juvenile idiopathic arthritis (JIA) is a rare condition affecting children. Like all patients, these children are told what treatment they need and then they are expected to comply. However, researchers in…
Brenda has both psoriatic arthritis and psoriasis. She's lived with the conditions since she was a child, learning to care for herself and deal with the challenges that arose. By…
The Sleeping Bear Dunes National Lakeshore Park, located in Michigan, has launched an innovated program to make their park more accessible to those living with a disability. They call it…
The Mental Health Rare Genetic Disease Network (MHRGDN) is a newly launched system, developed by the National Institute of Mental Health. The MHRGDN spans 15 research sites across the country…
Cyxone has just announced that the first person has been dosed in their Phase 1 clinical trial for Multiple Sclerosis (MS). This trial is investigating the effect of T20K. It…
No Cure, No Pay Emile Voest, an oncologist in the Netherlands, conceived the idea of a no cure, no pay model. After witnessing the exorbitant cost of drugs, some of…
Femtech Femtech refers to any type of technology that is centered on women's healthcare needs. This includes wearable breast pumps, apps to increase pelvic floor strength, menstruation apps, ovulation apps,…
For rare disease patients who face physical disabilities, even seemingly simple tasks can pose extreme difficulties. For instance, changing the channel on the television. Comcast Corp. previously created a voice…
The Study Juvenile idiopathic arthritis (JIA) is a rare disease that causes inflammation in the joints. It is of unknown origin and has no cure. Previous investigations in studies with…
Zogenix has just announced that they have not only finished enrollment in their Phase 3 investigation of Fintepla for Lennox-Gastaut Syndrome (LGS), but that the final patient has been randomized…
Eurordis-Rare Diseases Europe Eurordis-Rare Diseases Europe is an alliance of patient organizations which works to give rare patients a voice. It also strives to spread awareness of rare diseases to…
Heather White Heather White is a 41-year-old woman who was diagnosed with common variable immune deficiency (CVID) back in 2004 after fighting frequent bouts of pneumonia. Doctors believe that she…
This is the story of a brave journalist who was determined to improve care for rare disease patients in her country, North Macedonia. North Macedonia is a developing country located…
Dinesh Khanna, a professor at the University of Michigan has worked heavily with the rare disease scleroderma. Michigan has its own Scleroderma Program, of which Khanna is the director. Through…
Gaucher disease (GD) is a rare lysosomal storage disorder. It is caused by a deficiency in the glucocerebrosidase enzyme. In June, a new study was published in the International Journal of…
Growing up "Normal" Growing up, Steph Derham always thought she was normal. She says she has her parents to thank for that. Steph is incredibly grateful that her mom and…
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