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…
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…
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…
In 2022, only around 63 cases of ECHS1, an ultra-rare metabolic disorder, were reported around the globe. A study published in the Annals of Clinical and Translational Neurology in 2015…
The complexity of rare diseases makes communication between adults involved with the care of these children a vital tool. Communication and mutual trust are of the utmost importance. About the…
JScreen is a non-profit organization headquartered at the department of Human Genetics at Emory University in Atlanta. JScreen offers genetic testing and educational services, such as genetic counseling. JScreen's mission…
A mother knows best. In Kaitlyn Fryar's case, her intuition about her son Hudson led her to pursue help from numerous doctors. According to CBS Pittsburgh, Hudson seemed to…
On March 9-10, 2023, Dynamic Global Events hosted the virtual Global Innovation in Patient Advocacy conference. This event was geared towards various stakeholders in the rare disease space and was…
In many rare conditions, early diagnosis and treatment typically correspond with better outcomes. Prior to March 2023, LIVMARLI (maralixibat) was approved for the treatment of cholestatic pruritus related to Alagille…
The Mississippi State University (MSU) Bulldogs joined forces with Children’s of Mississippi, the state’s only children’s hospital with statewide pediatric clinics, to honor pediatrics patients throughout Mississippi, sharing and uplifting…
Stacey and Jason Chappell love their large family; there’s nothing they enjoy more than spending time with their five children, sometimes even going on fun vacations. But Stacey feels nonplussed…
Receiving a diagnosis can be difficult, and when you add into the equation that it’s for a rare disease, it can be a very lonely and isolating time. There is…
As reported by Valley News Live, Senator Amy Klobuchar (D-MN) has long been a supporter of the rare disease community. In the past, Senator Klobuchar has spoken at virtual press…
Nathan Barnica has always held a keen interest in and passion for bowling. He has honed his skills over the years through involvement in different team leagues. But in 2018,…
At the end of February 2023, workers at the Escanaba Billerud Paper Mill began feeling ill. Their symptoms were reminiscent of atypical pneumonia; some people struggled to breathe. According to…
Raising rare disease awareness is incredibly important - not just to spur research, but to validate the experiences of those within this community. Around Rare Disease Day on February…
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…
According to a story from sciencex.com, Queen's University Belfast, University College Dublin, and a team of 33 other partners have come together to start the All-Ireland Rare Disease Interdisciplinary Research…
For the first two years of his life, Rafe Hibben seemed to be progressing like a relatively healthy little boy. But when he was two years old, Rafe’s health went…
Could an ocular implant be an effective therapeutic solution for individuals living with noninfectious uveitis? According to an article in HCP Live, the answer seems to be yes. A study…
For the last eight years, the National Organization for Rare Disorders (NORD) has developed a State Report Card; the goal of this project is to evaluate the effectiveness of…
Lia Porcano was born in March 2018—and her parents Rosalyn and Justin could not have been more excited. But when Lia’s newborn screening test came back abnormal, the parents’ concern…
There are many rare or congenital diseases that, without early detection and treatment, can cause serious health issues and complications. For example, early treatment of homocystinuria (HCU) could delay or…
Amber Freed has long been a tireless advocate for the rare disease community. After two years of IVF, she became pregnant with her two children: Maxwell and Riley. About four…
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