According to a story from news-medical.net, a recent study has a found that a hidden disease called hemochromatosis could be a major risk factor for several different joint diseases and…
UPI Health News recently reported that a study, published in the New England Journal of Medicine, showed that fecal microbiota transplantation resulted in a 32 percent remission rate in patients…
Bontulinum Neurotoxins (BoNTs) are a protein that are used to treat a wide array of conditions including cervical dystonia, blepharospasm, neurogenic detrusor overactivity, and sialorrhea to name a few. These proteins…
The development of gene therapy as a treatment for rare and life-threatening conditions is a quickly growing area of scientific exploration. As more drug developers have begun investing in this…
Cystinosis Cystinosis is a rare disease which is caused by a buildup of cystine in the body's cells. This buildup impacts various organs including the brain, eyes, liver, kidneys, and muscles.…
Mucolipidosis Type IV Mucolipidosis Type IV (ML-IV) is a rare disease which causes a reduced mental capacity and blindness. ML-IV is caused by a missing or malfunctioning TRPMLI protein. Most patients never…
According to a story from bioengineer.org, it has long been known that people with the rare disorder Duchenne muscular dystrophy are at a greater risk of developing a rare cancer…
Spinal muscular atrophy (SMA) is a genetic condition which affects between 10,000 and 25,000 in the United States. While rare, it is currently the leading genetic cause of infant mortality. But,…
Nonalcoholic fatty liver disease (NAFLD) is a condition which causes fat to accumulate in the liver. Nonalcoholic steatohepatitis (NASH) is a severe form of the disease in which the patient also…
The government shutdown hasn't had any startling affects on rare disease research yet, but drug developers have voiced their concern that it could soon begin to hinder the research process.…
Xconomy.com has just released that funds have been raised for a variety of companies investigating therapies for rare conditions. Unfortunately, novel therapies and inventions cost million of dollars to develop. Thankfully,…
Myelodysplastic syndromes (MDS) is the name used to describe a group of stem cell disorders in which the body can't produce an adequate amount of platelets and blood cells. Currently,…
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…
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 news-medical.net, a team of researchers from UCLA have successfully developed an approach that allows for the reversal of the mutation that is responsible for the…
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 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.…
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 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 Business Wire, Basilea Pharmaceutica, Ltd., a partner of ArQule, Inc., recently released interim results from its Phase 2 clinical trial testing the company's investigational drug…
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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