Last year, Takeda, Eurordis, and Microsoft announced new collaborative efforts in the realm of rare disease. Specifically they were focused on the diagnostic period of rare conditions. However, up until…
Music and rock 'n' roll lovers already know him name, but Peter Frampton soon will become a new type of rock star: rare disease advocate. The British guitar legend announced…
Tommy Salisbury is eighteen years old. Unlike many teenagers, he is making big moves in the fundraising community and working hard to be a leader in efforts to find…
What is Pulmonary Arterial Hypertension? Pulmonary arterial hypertension (PAH) is a rare form of high blood pressure. Normal blood pressure is around 14 mm HG at rest. Someone with PAH…
According to a publication from BioSpace, the French biopharmaceutical company Sanofi has successfully secured FDA approval for their acquired thrombotic thrombocytopenic purpura (aTTP) drug Cablivi. This represents the first approved…
A traditional Dupuytren narrative is that Dupuytren is primarily a painless nuisance problem of bent fingers in old men. This perception does a disservice to many. Not only is Dupuytren common…
Researchers at BGN Technologies (at Ben-Gurion University) have come up with a new way to predict and monitor progression of a wide array of neurodegenerative diseases. One of the illnesses…
According to a story from Food Safety News, an illness known as chronic wasting disease has been known to affect members of the deer family in the US since the…
According to a story from the Huffington Post, a recent Instagram post from the Barbie brand of dolls indicates that the company is beginning to expand its line of dolls…
According to a story from Healio, wearable technology that is meant to help users monitor their health and fitness have continued to grow in popularity in recent years. Most people…
According to a story from BioPortfolio, the biopharmaceutical company Complexa, Inc. recently announced that it has dosed the first patient in its Phase 2 clinical trial which is testing the…
According to a story from PR Newswire, the specialty pharmaceutical company Mallinckrodt plc recently announced the release of interim data from the company's Phase 3 clinical trial. This trial is…
What is the Patient Advocate Foundation? The Patient Advocate Foundation (PAF) is a non-profit, formed in 1994 that has one primary aim- eliminate the barriers to treatment too many patients…
According to a publication from Genetic Obesity News, a new genetic mutation has been discovered in an Iranian family that has differing affects on early-onset obesity based on sex. Leptin,…
Happy Wednesday! Today, we're highlighting an article about gene therapy for MPS, followed by a story about a singer with Achalasia. After that, we have an article about app for…
A grandmother of four in Maryland has recently become the first cancer patient to complete the experimental CAR-T cell procedure at Georgetown University Hospital. Daisy Diggs 67, who was…
What is Precision Medicine? In essence, precision medicine refers to the idea that in order to establish the best treatment plan for a patient, we must look at them as…
According to a story from prnewswire.com, the biopharmaceutical company Neurotech Pharmaceuticals recently announced that the company had earned Fast Track designation from the US Food and Drug Administration (FDA). This…
The first step in treating rare diseases is accurately diagnosing them. Unfortunately far too many people go years without knowing what is going wrong in their body. Doctors are trained…
According to a story from BBC, Mats Steen passed away at 25 years old due to complications from his Duchenne muscular dystrophy. Upon his death, parents Robert and Trude grieved…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. This forum will take place in Jacksonville, Florida When: Saturday, March 30th, 2019 Where: Mayo Clinic Cancer…
Our partner, The MDS Foundation is sponsoring another free MDS Patient & Family/Caregiver Forum. This forum will take place in Phoenix, Arizona. When: Saturday, March 9th, 2019 Where: Sheraton Crescent…
The Washington Post reported in December of last year that the 8-year-old Matadi Sela Petit had died during surgery in a Los Angeles hospital. The accident was caused by a…
According to a story from EurekAlert!, a new type of cancer drug that is being described as a "Trojan horse" that infiltrates cancer tumors is showing potential in several types…
Cryptococcosis Cryptococcosis is a disease caused by the Cryptococcus fungus. It can affect the lungs, the spinal cord, or the brain. When the brain is infected, the condition is called…
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