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…
Allergan and Paratek Pharmaceuticals Inc. recently tested a drug called sarecycline for treatment of severe acne. The medicine is a tetracycline-derived antibiotic that is taken orally, and the best part…
On June 21st, Targeted Oncology posted the results from a phase 2 clinical trial for chronic lymphocytic leukemia (CLL). The trial examined the effects of combing two therapies--obinutuzumab and bendamustine.…
Aeglea BioTherapeutics recently provided an update concerning their clinical trial for arginase deficiency disorder. The four-word summary? They still need patients. Aeglea specializes in amino acid metabolism. Their focus is…
Clinical trial facilitators waste so much time that could be spent on research, trying to find patients to participate in their studies. We all know time is valuable, especially in…
The U.S. Food and Drug Administration (FDA) is quite an impressive agency. They regulate the safety and security of all foods that are produced, imported, or created in any other…
A clinical trial is up for those suffering from Duchenne Muscular Dystrophy. An oral tablet called Idebenone will be compared to a placebo, to see if it is safe and…
Pharmaceutical companies Takeda and Ovid have agreed to work together to develop a new drug for rare pediatric epilepsies. The continued development of Takeda’s compound TAK-935 will be shared by…
There’s a CRPS clinical trial being listed on Craigslist. Not kidding. You can check it out here. Now, am I the only one who finds that really odd? When I…
For anyone living with severe chronic pain from Complex Regional Pain Syndrome (CRPS), the best possible gift they could receive—short of a cure—would be a good treatment. To date, however,…
Cancer may have been called the Emperor of all Maladies but FOP-Fibrodysplasia Ossificans Progressiva is certainly the King. We place it lower only because it affects so few; one in every…
Fabry Disease is the number number two Lysosomal Storage disease in terms of numbers affected. It is an X-linked disease, with males passing on the mutated allele to all of their…
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