The very first patient in Medigene AG's clinical trial examining MDG1011 has officially received the treatment. This is the company's first human clinical trial for this TCR therapy. The trial…
Happy Rare Disease Week! This week has been all about rare diseases. Today, we're highlighting an article from a PW contributor with FAP, followed by a story describing some shortcomings…
Proteus Syndrome Proteus syndrome is a rare disease caused by the overgrowth of tissues including the skin, adipose tissue, and parts of the central nervous system. It also affects the…
According to a story from Gene42, a new rare disease platform called Genomics4RD has been launched in Canada. This is the first national research platform dedicated to rare disease to…
NICE stands for the National Institute for Health and Care Excellence in England. This organization is responsible for evaluating the cost effectiveness of all medications as they come to the…
According to a story from EurekAlert!, a recent study revealed that the surgical removal of the thymus gland, known as a thymectomy, was able to provide long-term benefits for patients…
According to a story from BioSpace, there are times when new information is learned about a medication or therapy outside of the precisely controlled environment of a clinical trial. Such…
According to a story from ALS News Today, ICD-10 disease codes will soon be up for revision in March 2019. What does this mean exactly? Well ICD stands for the…
How Liver Transplant Recipients are Selected Liver transplant recipients in the United States are selected according to their MELD score. This number is calculated from lab values. It aims to…
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…
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