Results from a Phase 3 trial of the medicine Lynparza (olaparib) for the treatment of advanced ovarian cancer in women with the BRCA-mutation have been announced as positive. The full…
According to a story by CBS News, an unsuspecting virus may become a potential treatment for brain cancer. The study showed that those who were given a genetically modified version…
Happy end-of-June, readers! A lot of people are getting ready for the fourth of July right now. However, as a lot of the rare community knows, not everyone is able…
According to a story from news-medical.net, a team of scientists from Delaware have recently released a study in the journal BMC Bioinformatics. In this study, the researchers found that patters…
Researchers have been studying an experimental treatment for cystic fibrosis that is thought to be effective regardless of a patient's genetics. The research is still in the early stages, but…
My dysautonomia story starts around the year 2000, when I was 13 years old, though I never got a diagnosis until I was about 28! In the Fall of 2000, I…
According to a story from Reuters, the drug development company Merrimack Pharmaceuticals decided to abort its development of a new treatment for pancreatic cancer. The company chose to let go…
According to News Medical, a group of researchers at Johns Hopkins and the Bloomberg-Kimmel Institute for Cancer Immunotherapy recently found encouraging results for a study involving immunotherapy to treat a…
Amicus Therapeutics is developing a treatment for Pompe disease through its AT-GAA program. They have recently announced that they have met with regulators from the European Union and Germany who…
June is Apert Syndrome Awareness Month! As summer sets in, let's take a moment to learn a little more about this rare disease and do our part to raise some…
According to a story from globenewswire.com, the company Revive Therapeutics, which is dedicated to the development of treatments derived from cannabis and cannabinoids, recently announced that the FDA has decided…
According to a story from bioengineer.org, a group of scientists working at the Columbia University Irving Medical Center (CUIMC) have managed to develop a computational model that could potentially make highly…
According to a story from news-medical.net, a group of scientists from the Cincinnati Children's Cancer and Blood Diseases Institute have recently published a study in the journal Nature Cell Biology. This…
According to a story from Wink News, an alternative chemotherapy approach is helping patients get rid of cancers of internal organs or otherwise in the abdominal cavity. The approach is…
Jim and Melea Martin are Outreach Partners with Future by Design, a family support group organized by the Cystinosis Research Network. Melea has cystinosis and Jim is her husband. We…
This article discusses the plot of the biographical film Brain on Fire. Susannah Cahalan was twenty-four when she began experiencing seizures, hallucinations, and, after only a few weeks, catatonia. Her…
Tomorrow is World Scleroderma Day! This date is an opportunity for anybody with an interest in scleroderma to join together as one united voice to shine a spotlight on a…
According to a story from triblive.com, a recent study demonstrated that Lyme disease cases increased at an "exponential" rate in the ten year period from 2003 to 2013. The area…
According to uk.reuters.com, the pharmaceutical company Sanofi is playing catch up to put itself ahead of the competition in developing the new cancer treatments. The French company didn't join in…
The National Society for Phenylketonuria (NSPKU) is holding their PKU Diet Challenge today. The diet challenge is specifically aimed at MPs (Members of Parliament in the UK-- aka, not me),…
According to a story from today.com, Dr. David Fajgenbaum was first diagnosed with Castleman disease in 2010. His first encounter with the illness was terrifying. He was confined to the…
Understanding diseases and the mechanisms behind them is a crucial step towards developing treatment. Many times, however, it's difficult to accomplish this due to the rarity of a condition, or…
I had the chance to chat with Amber Olsen, the founder of The United MSD Foundation, and mother to Willow. This is their story. Willow had been a seemingly healthy…
Visit any pediatric ward in a hospital or medical center and you will see many pieces of medical equipment, but visit the OT and PT departments and you are in…
According to EurekAlert! Science News, researchers have pinpointed the certain immune cell that further inflames a heart attack. The immune cell, however, comes with good intentions of trying to fix…
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