According to a study published on March 22, 2023 in the scientific journal Frontiers in Endocrinology, the rare genetic disorder X-linked hypophosphatemia (XLH) has a negative impact on the function of…
The International Congress of Parkinson's Disease and Movement Disorders took place this year from August 27 to 31. During the Congress, researchers and other stakeholders from across the globe…
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…
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…
When a family member is unable to care for themselves, proper training is essential for all parties involved. A case in point was the crisis Patti LeFleur was unable…
65 million people in the U.S. who are on Medicare are prescribed medications according to a mid-year KFF healthcare survey. 60% of these individuals are prescribed a minimum of…
NOTE: The article was originally published as a blog post from the BHD Foundation a nonprofit patient advocacy organization. Birt-Hogg-Dubé syndrome (BHD) is a rare inherited condition associated with changes…
Teegan is Kelly and Matt Olson’s youngest daughter, so when she began missing developmental milestones, her parents were on alert. After a variety of tests over the span of…
Accutar Biotechnology, Cranbury, New Jersey recently announced via BioSpace that dosing has begun with the first patient enrolled in the Phase 1 trial of AC0676 a chimeric degrader molecule…
According to a story from Healio, a recent study doesn't bode well for people living with hidradenitis suppurativa (HS), a serious and debilitating skin disease. The data found that comorbidities…
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…
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…
According to a press release from Verrica Pharmaceuticals and its partner Lytix Pharmaceutical via Healio, results of Part 1 of the phase 2 study of their investigational therapy candidate VP…
A 15-year search for a diagnosis by Lilly Grossman and her parents ended in 2013. Lilly became the first person in the world to be diagnosed with ADCY5-related dyskinesia.…
Currently available therapeutics may not be adequately effective in the fight to treat late-stage acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). Researchers and drug developers are working to identify…
According to a story from Axios, the relatives of Henrietta Lacks are filing a second lawsuit against a biopharmaceutical company, alleging that the company is unfairly profiting through the use…
Galila Yohannes’ parents, who originally hail from Eritrea, brought their family to Israel for a better life. And while they never would have expected what came next, this move likely…
According to a story from Medpage Today, a study conducted by scientists at the University of Texas MD Anderson Cancer Center highlighted the substantial benefits of aerobic exercise in people…
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…
An Intern’s Journey When my summer internship with Red Nucleus began, I had little familiarity with rare diseases and the life science industry. A distant relative of mine struggled with…
The U.S. FDA has approved two treatments for people living with idiopathic pulmonary fibrosis (IPF). These therapies—Esbriet (pirfenidone) and Ofev (nintedanib)—both work to reduce fibrosis, or scarring, in the…
In Jordyn Sava's reporting from Targeted Oncology, readers learn that the U.S. Food and Drug Administration (FDA) granted clearance to a global Phase 3 study. This means that the…
Unfortunately, gaining access to care within the rare disease space can be difficult. There is often lesser education and awareness about rare conditions, less research performed, and poorer access to…
21-year-old Chloe Feighery has a deep love and appreciation for her younger sister Heidi, who is just one and a half years old. When she learned that she was becoming…
Soon after Emma Young was born, she was diagnosed with a rare genetic disorder called Bannayan-Riley-Ruvalcaba syndrome (BRRS). At two months old, she battled COVID-19. Then she was diagnosed with…
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