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…
The Star Gazette recently reported that Alan Ramsay Evans, a 34-year-old man from Horseheads, New York, remains in the hospital after developing respiratory failure. Three decades ago, when he was…
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…
What is Distal Renal Tubular Acidosis? Distal renal tubular acidosis (dRTA) is a rare condition which affects approximately 1 in every 100,000 people worldwide. It is caused by a build up…
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…
According to a story from Oncology News, a team of researchers affiliated with UNSW Sydney have made a discovery that could have major implications for the rare cancer neuroblastoma, a…
According to a story from Juvenile Arthritis News, a recent study shows that a greater percentage of children with juvenile idiopathic arthritis are participating in school sports activities in the…
The typical treatment for Fabry disease is Fabrazyme which is an enzyme replacement therapy (ERT). This therapy must be administered to the patient every two weeks. A new ERT created by…
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…
Passsage Bio Passage Bio has just announced that they've received 115.5 million dollars to help advance their development of gene therapies. They will begin by using the funds to further…
Cystic Fibrosis Cystic fibrosis (CF) is a rare condition that causes mucus to build up in the lungs and the digestive system. It results in chronic lung infections, lung inflammation, and…
Langerhans Cell Histiocystosis Langerhans Cell Histiocytosis (LCH) Is a rare form of cancer caused by an excessive amount of Langerhan cells in the body. These cells form granulomas which can have a…
Fetal Surgery for Spina Bifida Spina bifida is a rare condition which results in improper spine formation during embryonic development. A gap occurs in the vertebrae causing nerve damage in varying…
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,…
Hydrocephalus Hydrocephalus is a rare disease which causes a build up of fluid in the ventricles of the brain. The continuous flow of this fluid is how the body prevents the…
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…
According to a story from Central Charts, the biopharmaceutical company Inventiva recently announced the release of results from a Phase IIB clinical trial which tested the company's lead product candidate,…
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