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…
Sometimes, clinical trials do not go the exact way that is expected. According to Targeted Oncology, this is what happened in a Phase 1b clinical trial evaluating RVU120 for…
A survey of oncologists has found that more education is clearly needed regarding cholangiocarcinoma. Specifically, more education would be beneficial regarding testing and targeting the mutations which cause the condition…
Bacterial meningitis is a severe condition that is linked to high mortality. Unfortunately, newborns are much more susceptible to bacterial meningitis. In fact, this infection occurs more than 30 times…
According to a story from MSN, mother Vanessa Ruiz is facing a terrifying choice: feed her family or pay for the genetic testing to confirm that her children Faith and…
Having a rare, chronic disease can impact one's life significantly, whether it's physically, spiritually, socially, or psychologically. Not only this, but those responsible for these patients' care are affected as…
Becca Meyers, a 26-year-old swimmer who was born deaf, has just been told she can't bring her care assistant to the Tokyo Games. As a result, she has had to…
On July 21, 2021, Patient Worthy attended an online webinar presentation titled "How RDCA-DAP Can Help Inform Optimal Trial Design in Progressive Rare Disease." Organized by the Critical Path Institute…
The European Medicines Agency (EMA) recently received a Marketing Authorization Application (MAA) for Mycapssa, a maintenance therapy for acromegaly. Chiasma submitted the application, basing it on the results of their…
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 recent article in Genetic Engineering News, findings by scientists at the University of Boston’s School of Medicine and Massachusetts General Hospital (MGH) have uncovered two plausible…
According to a video from OncLive, Dr. John P. Leonard from Weill Cornell Medicine discussed the role of PI3K inhibitors in the treatment of follicular lymphoma, a rare cancer. While…
Unfortunately, there are a great deal of inequities within the healthcare system. Health inequity occurs when groups are treated differently, offered different or worse care, or have a lower…
According to a press release from July 15, 2021, the Chinese NMPA recently approved an Investigational New Drug (IND) application submitted for CAN106, a potential therapeutic option for patients with…
According to a press release published on Globe Newswire, the biopharmaceutical company Saniona has announced recently that its investigational therapy Tesomet has recently earned Orphan Drug designation from the US…
A better comprehension of one's cancer can provide improved treatment outcomes. It's easier to fight something that one understands. That's why a recent article published in Medical Xpress is so…
Near the end of June 2021, pharmaceutical company Crinetics Pharmaceuticals, Inc. ("Crinetics") shared via news release that the first patient had been dosed in the Phase 3 PATHFNDR-1 clinical trial.…
Medical studies are crucial players in the journey to develop new and more efficacious treatment options for patients with rare diseases. On June 10, 2021, Vertex Pharmaceuticals Incorporated ("Vertex") shared…
Written by: Ashley Walker Like many couples, my husband Johnny and I felt that the birth of our twin sons would mark the beginning of our family’s story. We certainly…
Underserved communities have difficulties when trying to access the resources that are available to the majority. Because of this, more work has to be done to ensure that everyone can…
The FDA has recently approved KEYTRUDA (pembrolizumab) in combination with LENVIMA (lenvatinib) for the treatment of advanced endometrial carcinoma that is not mismatch repair deficiency or microsatellite instability-high. This approval…
The symptoms of terminal pancreatic cancer are subtle. The disease is usually not diagnosed until it has substantially progressed, leaving a dismal five-year overall survival of five to ten percent.…
In the past, researchers have shown that acute myeloid leukemia (AML) may be driven by certain genetic mutations. For example, some mutations might cause the abnormal or excess production of…
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…
Genetic editing has the potential to address and treat a variety of genetic conditions. Recently, researchers explored gene editing as a potential therapeutic option for patients with facioscapulohumeral muscular dystrophy…
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