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…
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