It all began with an email in 2019. Matt Might, director of the Hugh Kaul Precision Medicine Institute, shot his thoughts over to neurosurgery resident Matt Davis, whose 3-year-old son…
Why are rare diseases so difficult to study? Is it just their rarity? Or is it also industry challenges? Unfortunately, industry has a lot to do with it. Clinical Trial…
Big development for those in the primary hyperoxaluria type 1 (PH1) community! Alnylam Pharmaceuticals just announced that its Phase 3 study of lumasiran, an investigational therapy for the treatment of primary…
Some promising news for the congenital adrenal hyperplasia (CAH) community! Spruce Biosciences is one step closer to a treatment for CAH after new phase 2a data showed the drug reduced…
Eosinophilic Esophagitis (EoE) Eosinophilic esophagitis (EoE) is a rare and chronic disorder which affects the digestive system. Eosinophils are a type of white blood cell that help fight infections. This…
According to a publication from BusinessWire, the Maryland-based pharmaceutical company OncoImmune, Inc. has announced successful phase 2 trial results for its acute graft versus host disease (GVHD) drug CD24Fc. Graft…
Cold agglutinin disease (CAD) occurs when the body's antibodies destroy their own red blood cells. It is a form of hemolytic anemia, meaning the body can't produce enough blood cells fast…
Prader-Willi syndrome (PWS) is a neurodevelopmental disorder which causes intellectual disability, a deficiency of growth hormones, compulsive behavior, a high risk of obesity, and hyperphagia, a symptom which can be…
There's some good news for the hereditary angioedema patient community this week! Shire, a global biotechnology company focused on rare diseases, announced in the Journal of the American Medical Association (JAMA) publication…
Zealand Pharma A/S (Zealand) recently announced in a press release the start phase 3 trial for short bowel syndrome treatment. The trial investigates the use of glepaglutide as a treatment…
A report on newsmedical.net details a major grant awarded for the research of idiopathic pulmonary fibrosis (IPF). The grant totals $8.9 million, aiming to fund a program for several years.…
A new clinical trial aims to be the first treatment for mitochondrial disease to be granted FDA approval. Whether or not the trial results in a landmark treatment, it is…
Major pharmaceutical company Pfizer recently announced the success of a new experimental product. The drug targets a disease called transthyretin cardiomyopathy, related to amyloidosis, a rare and dangerous disease that affects…
A phase three clinical study is beginning for a new glioblastoma treatment. The new drug is called VAL-083 and is specially designed for treatment of late-stage glioblastoma. The study will…
As parents, we want to protect our children from harm and keep them safe. So when your child is suffering from a rare metabolic disorder, it's natural to feel helpless.…
A Futurism article this week put into very human terms the ongoing question we face when it comes to rare disease treatment and Pharma: What is the cost of life?…
The FDA this week granted orphan drug designation to glepaglutide for the treatment of short bowel syndrome (SBS), developed by Zealand Pharma A/S. SBS is a rare disorder where an…
At first glance, little Victoria Thompson looks like your average happy and healthy toddler. You'd have no idea that once upon a time, she had a tumor on her kidney…
It's called Hereditary multiple exostoses (HME). Yes, quite the mouthful. Our friendly neighborhood scientists at Children's Hospital of Philly (CHOP) have been working diligently to study this rare bone disease…
Cancer treatment, much like golf, is all about the follow-through. It is true that chemotherapy and surgery are important. However, it’s the maintenance therapies that keep people living for years…
Cancer. This word can inspire fear in even the most stalwart of individuals. How many other diseases are known by a phrase involving just the first letter? The Big C.…
All you have to do is look around. If you do, you’ll see the rate of progress accelerating. Look at anything modern. Some people liken this to Moore’s Law concerning…
Most things have multiple functions. The tire iron you use to jack up the car when changing a flat tire? It can be used as a self-defense weapon if an…
If you’re a science nerd, we live in some pretty exciting times. Even though we’re going through a moment where it feels like science and empirical evidence itself is under…
At some point in the past, we decided that it would be important to divide things up when we study them in school. For a long time, all science was…
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