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…
At the end of June 2021, pharmaceutical company Harmony Biosciences Holdings, Inc. ("Harmony") shared the initiation of a Phase 2 clinical trial via news release . During the trial, the…
In the European Union (EU), Orphan Drug designation is granted by the European Medicines Agency (EMA). The status is given to drugs or biologics intended to treat, prevent, or diagnose…
In the United States, the Orphan Drug Act was developed to expedite the development and review of drugs or biologics intended to treat rare or life-threatening conditions. For the purpose…
The FDA has recently granted their approval to a Phase 2 trial of FHL-301, a treatment for those in the earlier stages of Parkinson's disease. Forest Hills Lab will conduct…
The primary cause of death in melanoma patients is widespread metastases. Currently, there is no cure in cases of relapse or in advanced stages. A recent article in Science…
The European Commission recently approved of Enspryng for neuromyelitis optica spectrum disorder (NMOSD). Now, European patients have a new treatment option. This one is especially useful as it can be…
According to a story from Parkinson's News Today, a small-scale study suggests that Virtual Reality (VR) gaming could be a useful and effective form of exercise for people living with…
As reported in World Pharmaa Today, Valneva SE is a specialty vaccine company in the business of the creation and distribution of prophylactic vaccines that treat infectious diseases with serious…
About a month ago, from June 23-26, The Immune Deficiency Foundation hosted the 2021 Primary Immunodeficiency Conference. The event was the largest gathering of primary immunodeficiency (PI) patients across the…
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