The passage of the Orphan Drug Act was an important step in creating an environment that supports rare disease research and drug development. This Act created Orphan Drug designation,…
According to a story from BioPharma Dive, the drug company AbbVie has recently announced its decision to withdraw two different approvals for its cancer therapy Imbruvica. The approvals were withdrawn…
When Al and Terry Rezsel were married, they took their vows seriously: in sickness and in health. So when Terry was diagnosed with breast cancer in 2010, after 10 years…
The International Association of Fire Fighters (IAFF) shared in March 2023 that firefighters in Ontario would now have presumptive pancreatic cancer and thyroid cancer coverage. Presumptive coverage means that…
Have you ever heard of the Orphan Drug Act? This Act helped stimulate and advance the development of, and research into, therapeutics for individuals living with rare or underserved conditions. In…
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…
Historically, mesothelioma has been difficult to treat. Although treatment options exist, the cancer tends to respond poorly which results in a higher mortality rate. Improving patient care and outcomes (such…
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…
When asked about what information they wished they were given during their medical journeys, survivors of thyroid cancer reported a desire to have had more information on treatment. An article…
According to a press release from the US Food and Drug Administration (FDA), on March 24, 2023, the agency issued a new draft guidance titled Clinical Trial Considerations to Support Accelerated…
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…
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…
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…
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…
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…
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…
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…
The 1980 World Series was a grand event for the Philadelphia Phillies. It was the last inning, and the Phillies were up by 3. With two players out and…
While the five-year survival rate for adenoid cystic carcinoma (ACC) is relatively high, this cancer has a high risk of recurrence. Therapeutic options are needed to manage this cancer and…
In early stages, renal cell carcinoma (RCC) is treatable and has a high five-year survival rate. However, if the cancer progresses or metastasizes (spreads), the five-year survival rate falls…
Currently, an estimated 47-50% of global nasopharyngeal carcinoma cases occur in China, with the regions of Guangdong, Guangxi and Fujian being heavily affected. There are a number of treatment…
Cases of multiple myeloma seem to be rising across the country and the globe. New and effective therapeutic interventions have the potential to significantly benefit this patient population. Ichnos Sciences…
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