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…
At Patient Worthy, we seek to partner with patient advocacy and other related organizations in order to collaborate and promote one another's activities. Recently, we have begun a partnership with…
When Harper Webb was just six weeks old, her mother Johnna grew concerned about the girl’s health—call it mother’s intuition. Harper struggled with sleeping and feeding. Johnna began taking Harper…
Certain patients with ulcerative colitis (UC) do not respond well to steroid treatment. These patients are considered to have steroid-refractory acute severe ulcerative colitis, or ASUC. Other therapeutic options, such…
Approved for use in 2017, emicizumab—marketed under the brand name Hemlibra—has shown immense benefits in the treatment of hemophilia A. But researchers wanted to better understand how emicizumab, used…
On March 23, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
April 8, 2023 will be recognized as Cushing's Awareness Day, a time to help spread awareness among the general public and the medical community about Cushing's syndrome and Cushing's disease,…
Written by Jenny, Life's a Polyp I come from a long family line of individuals with familial adenomatous polyposis (FAP). My family though would learn the most about FAP because…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
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 families,…
Prior to the end of March 2023, ganaxolone had received six separate Orphan Drug designations from the FDA; more recently, as shared in a news release from commercial-stage pharmaceutical…
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