The National Institutes of Health (NIH) held its in-person Rare Disease Day at NIH convergence on February 28, 2023. This was the first time in a while that the event…
Reanna and Malaky always knew they wanted to start a family together. They tried for a while without success—so they were thrilled when they learned that Reanna was pregnant with…
According to a press release from the US Food and Drug Administration (FDA), on March 24, 2023, the agency issued a new draft guidance titled Clinical Trial Considerations to Support Accelerated…
People living with bronchiectasis, a chronic condition characterized by damaged airways, experience progressive lung damage and inflammation. This can lead to a multitude of consequences, including a reduction in…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
STATHMIN-2 (STMN2) is a protein that has been proven to play a role in axonal development, repair, and stability. This protein is highly expressed in human motor neurons; research…
Currently, the standards-of-care for narcolepsy include lifestyle changes involving diet and exercise, behavioral therapy, nap therapy, and certain medications designed to combat excessive daytime sleepiness. However, a majority of…
Boshamps Seafood & Oyster House has been serving Destin, FL with authentic Gulf seafood, well-mixed libations, and live music for years. But that isn’t the only way Boshamps has made…
Data from the first cohort of the Phase 2 KEYNOTE-158 trial, evaluating pembrolizumab for individuals with advanced thyroid carcinoma, showed positive safety and efficacy. According to Cancer Network, 103 participants…
The Orphan Drug Act of 1983 was designed to facilitate the development of orphan drugs, or drugs intended for rare or life-threatening indications. In the United States, rare conditions are…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Being diagnosed with a rare or chronic illness can come with a heavy emotional and psychological burden. When you first receive a diagnosis, you might be wondering what it…
As many parents within the rare disease and chronic illness community know, it can sometimes be difficult to parent a medically complex child. It is important to practice self-care…
According to reporting for Cure Today, the FDA recently granted Accelerated Approval to a combination treatment of Padcev (enfortumab vedotin-ejfv) and Keytruda (pembrolizumab) for advanced or metastatic urothelial carcinoma.…
Cases of pancreatic cancer have been increasing throughout the United States over the past few decades. Although the 5-year survival rate recently rose to 12%, pancreatic cancer is on track…
For the first year of his life, Celebi Remillard and his mother Tatyana frequently took the drive from Klamath Falls to Portland to visit his care team. When Celebi was…
In the United States, Orphan Drug designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics that are being developed for rare conditions. Rare…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
The Ovarian Cancer Research Alliance stated in new guidance that evidence indicates the majority of ovarian cancers begin in fallopian tubes. The president of the alliance, Audra Moran, was…
The Higbee family never planned to become rare disease advocates. But when their daughter Harlow began experiencing health issues, they knew that they would do whatever they could to help.…
When Sue Hitchmough first began feeling ill, she wasn’t sure what to think. Doctors told her that she had an ear infection, but antibiotics didn’t seem to fix anything. She…
Children with cystic fibrosis (CF) born between 1995 and 1999 had an estimated life expectancy of 32 years. Advances in both research and treatment have contributed to increased life…
Statins are a type of drug that can help you to lower your blood cholesterol levels. These drugs work by inhibiting HMG CoA reductase, an enzyme that the body…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In the past, there have been specific genetic mutations associated with breast cancer: most often the BRCA1 and BRCA2 genes. Altogether, there are 15 genes associated with a moderate-to-high…
© 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.
