Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It's just been published that in 2018 they approved a…
While medical students may go into the profession dwelling on the lives they can save, the inevitable fact of the profession is that there will be times when they…
According to an article recently published by Forbes, one data scientist wants to explain why data science has proven to be slow at identifying particular biomarkers in medical patients. Imran…
As discussed in a recent article on PatientWorthy the FDNA has recently developed a new kind of algorithm which can diagnose rare genetic conditions such as Angelman syndrome, Williams syndrome, and Fragile X syndrome using…
"We want to have better ways to figure out who is non-adherent so we can focus our efforts better on those patients who may require more assistance or specific resources…
Perhaps the most difficult thing about rare disease research is that not only do the conditions affect only a small portion of the population, but like every diagnosis, they affect…
According to a story from PR Newswire, pancreatic cancer rates in the US are increasing by almost two percent each year. This puts the disease in position to become the…
Juvenile idiopathic arthritis, Lupus, and Malaria are diseases which currently have limited treatment options. But a new discovery by researchers at the Benaroya Research Institute (BRI) could potentially lead to…
According to a story from New Scientist, a team of scientists at the Institute of Cancer Research in London were able to develop an AI tool that was able to…
According to a story from BioPortfolio, the Bristol-Myers Squibb Company recently announced that the European Commission has recently approved the first combination treatment for renal cell carcinoma available in the…
For those with a complicated medical diagnosis, state-funded health insurance bodes little practicality. Patients living with rare and chronic disease must schedule visits to multiple specialty clinics and physicians, especially…
According to a story from BioPortfolio, the keto-medical nutrition company Disruptive Nutrition recently announced that the first patient had been enrolled in a research study which will test a unique…
According to a story from Onclive, combination therapies are likely to play an increasing role in the treatment of chronic lymphocytic leukemia. In this video, leukemia treatment experts discuss the…
According to a story from The Conversation, scientists have determined that sex plays a significant role in outcomes when it comes to treating the rare and deadly brain cancer glioblastoma.…
The Right-To-Try Law is a federal legislation which allows terminally ill patients the opportunity to use experimental drugs that don't yet have FDA approval. It's controversial at best, but some…
Merkel cell carcinoma (MCC) is a form of skin cancer. In the United States, about 2,500 people are diagnosed with MCC each year. The condition is treated with chemotherapy, immune…
According to a story from Eurek Alert!, a team of scientists affiliated with the University of Helsinki have successfully developed a new, more useful Hirschsprung's disease mouse model. This disease…
FDNA has created a new type of algorithm which may be able to help diagnose rare genetic conditions like Noonan Syndrome by a simple picture of a person’s face. What’s particularly…
According to a story from BioPortfolio, the pharmaceutical company Acer Therapeutics, Inc., recently announced the company's New Drug Application (NDA) has been accepted by the US Food and Drug Administration…
Every day the scientific community makes progress in the study of rare disease. Research in the field has advanced exponentially in recent years and we're starting to see more treatments…
Pliant Therapeutics, Inc., a biotechnology company specializing in developing and commercializing treatments for fibrotic diseases, announced on January 3, 2019 through PRNewswire, that it has initiated a Phase 1 first-in-human…
According to a story from BioSpace, the biopharmaceutical company Liquidia Technologies, Inc., has recently released interim safety data for its open label Phase 3 clinical trial testing LIQ861. This data…
According to a story from Medical Xpress, a collaborative team of scientists from UT Health San Antonio, the Mayo Clinic, and the Wake Forest School of Medicine, have recently released…
According to a story from USA Today, two 29 year old rare disease patients, Daru Smith of Chicago and Sarah McPharlin of Grosse Point Woods, Michigan, both received triple organ…
We tend to daydream about miracle cures for rare and detrimental conditions like ovarian cancer. Sometimes they exist, but often it's more of a battle to find the right combination…
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