30 Things About My Rare Invisible Illness You May Not Know By Debra Richardson 1. The illness I live with is: stiff person syndrome & insulin-dependent diabetes. 2. I was…
.jpg)
A medication that works for some patients with a rare disease may not be effective for others, even with the same disease. For rare diseases, this is important because sample…
About three million Americans are affected by inflammatory bowel disease. Over the years there have been many improvements to treatment for this condition, but there are still further advances that…
On April 28th, 2020, Acromegaly Community, a nonprofit organization that is dedicated to supporting acromegaly patients, will be hosting a patient focused drug development meeting (PFDD). These meetings are an…
More than 20 years ago researchers at Harvard University, led by professor Oliver Pourquié, discovered how the vertebra in chickens first form. Essentially, a 'tick' initiates the formation of a…
By Rachel Whetstone from In The Cloud Copy The Orphan Drug Act of 1983 was created to encourage the development of drugs to help people with rare diseases. Drug companies…
Epithelioid sarcoma is a rare form (accounts for <1%) of soft tissue sarcoma. Until now, there have been no treatments specifically for this patient population. But the FDA has just…
By Rachel Whetstone from In The Cloud Copy When Helisabed Romano was pregnant with her daughter, she got some bad news. The the developing baby had spina bifida, a defect…
AI Therapeutics is a biotechnology company that uses artificial intelligence to match drugs to indications. They do so through the use of their algorithm, Guardian Angel. This algorithm has…
According to a story from Medical Xpress, a recent study that draws on 30 years of maple syrup urine disease patient data has concluded that while significant improvements have been…
According to a story from Medical Xpress, a team of researchers from the medical school at the University of Massachusetts has revealed new information about the nature of neurodegenerative symptoms…
This is part two of the story, click here to read part one After searching for a new neurologist, we found one only 30 mins away that was willing…
Cancer patients often face adverse effects from their treatment. These effects typically need to be addressed by a doctor, whether they're nausea, rashes, or anything else. The issue is that…
For much of human history, many genetic disorders were accepted as incurable, their rapid onset bound to take patients' lives before they had the chance to be lived. However, the…
In the wake of Martin Luther King Jr. Day, those with rare diseases have vocalized some of the dreams that they have for the healthcare industry. With all of the…
Seizures are a common symptom of Angelman syndrome (AS) and the cause of these seizures was unknown for a long time. This lack of knowledge made it impossible to…
SELLAS has just announced that patient screening has begun for a Phase 3 trial for acute myeloid leukemia (AML). The trial is examining the effects of GPS for patients who…
According to a story from Michigan Medicine Headlines, Domino's Farms is the home of a treatment center with unique capabilities: the Michigan Medicine Comprehensive Wound Care Clinic. One of the…
As originally reported in CBC News, Canada is deciding on new parameters to set for medically assisted death, legalized in 2016. Since legalization, 6,700 Canadians have made use of the service,…
It has been evident to scientists for years that Alzheimer’s disease, which affects about five million people in the U.S., is caused by an accumulation of two proteins within…
According to a story from IFLScience, the US Food and Drug Administration (FDA) approved a total of 48 novel medicines last year. A further investigation of these drugs reveals that…
Focal segmental glomerulosclerosis (FSGS) is a kidney disease that often results in end-stage renal failure. A kidney transplant is the best way to treat this failure. The issue with this…
According to a story from the Irish Medical Times, Ireland's Health Service Executive (HSE) has recently announced the extension of a reimbursement agreement between the agency and the pharmaceutical company…
People with rare diseases struggle to get treatment. If you have one, you're probably familiar with the statistic that only 95% of them have treatment options. Rare disease treatments have…
According to a story from The Edge Markets, Dr. Lee Boon Chye, Malaysia's Deputy Minister of Health, has recently announced an initiative to develop a National Framework for Rare Disease,…
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.
