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…
Medical research is incredibly important, especially within the rare disease community. Rare conditions tend to be under-researched and under-funded; this can make it difficult not only to better learn…
In the past, there has been limited research exploring the presentation of melanoma in Asian American and Pacific Islanders (AAPIs). Recent research has sought to explore this relationship and…
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…
What is the actual prevalence of atrial fibrillation (AF) on a local, national, and global scale? While this question may seem straightforward, the answer is not. An estimated 33% of…
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…
Drug development within the rare disease space can be tricky. In many cases, there is a general lack of research and understanding around these conditions; as a result, many…
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…
In 2022, the U.S. Food and Drug Administration (FDA) examined the potential of approving palovarotene, a selective RARγ agonist, for the treatment of fibrodysplasia ossificans progressiva (FOP). However, the FDA…
In 2014, Adam Tate was a college student who was working as a scout leader. One day, he went on a trip with some others to go ziplining. However, his…
The Muscular Dystrophy Association (MDA) recently held its MDA Clinical & Scientific Conference in March 2023. During the conference, stakeholders in the community discussed research trends and clinical practices associated…
According to a story from LA Weekly, a lot of media coverage concerning cannabis has been largely positive in recent years. The medicinal value of CBD, for instance, has seen…
In November 2022, the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) approved Upstaza (eladocagene exuparvovec) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. The product, which…
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