In my home, everything is shared with our dogs. When eggs are made for breakfast, the dog’s eggs are served scrambled. When it’s time to exercise, we all head outside…
A recent survey of more than 150 idiopathic pulmonary fibrosis (IPF) patients demonstrates the invisible burden the rare disease causes. It is no secret that any rare disease has the…
Over the years, I've talked with dozens of people with Parkinson's disease (PD). Male or female, old or young, they tend to have one characteristic in common, even if they…
Another great breakthrough in research science! A new study found that an experimental drug combined with the traditional chemotherapy drug cisplatin successfully eliminated a rare salivary gland tumor and prevented a recurrence…
As autoimmune disorders become an increasing problem throughout the world, particularly in developed countries, there has been an increasing amount of research dedicated to finding a cure, and the role…
Recent studies have shown promising results that cannabidiol (CBD) helps relieve different forms of epileptic seizures like those from Lennox-Gastaut syndrome and Dravet syndrome. Lennox–Gastaut syndrome (LGS) is particularly severe, childhood…
In breaking news for the progeria community, biomedical engineers have just succeeded in growing miniature human blood vessels that mimic progeria, the ultra-rare genetic condition characterized by premature aging in…
If you’re anything like me, you probably don’t remember much about the work you did 20 years ago. And unless you’re a pack rat, chances are you can’t even lay…
Acute diagnosis is the Holy Grail for all illnesses and conditions. But what if you could predict the likelihood that you would develop a deadly disease and take steps to…
Gene therapy is rapidly shaping up to be the next stage in the evolution of treatment for a wide array of conditions. And it makes sense: rather than treat the…
Batten disease is one of the rarest diseases in the world. In fact, it only affects about two to four out of every 100,000 births in the United States, which…
BIG NEWS in the ALS community this week. As reported by NewsWise, researchers at the Mayo Clinic and St. Judes Children's Research Hospital have discovered the dysfunctional cellular mechanism behind…
According to some Finnish researchers, children with heterozygous familia hypercholesterolemia (HeFH) should use statins as a treatment with caution. Though they have proven to be able to lower lipid levels…
In very recent news, the biopharmaceutical company Chiasma, Inc. announced that it has come to an agreement with the US FDA regarding the design of a phase III clinical trial…
In a study at the University of North Carolina (Chapel Hill), researchers found that there may be a much more effective new way to isolate lung stem cells in order…
Recent findings from research done at the University of Pennslyvania indicate a new biomarker associated with a group of rare metabolic diseases called mucopolysaccharidoses (MPS). A biomarker is a measurable…
A new study shows that two specific compounds found in common sunscreen are actually effective as multiple sclerosis symptom suppressors. These two substances are salate derivatives and are also part…
A North Carolina-based research group has just developed research that indicates a potential stem cell treatment for lung fibrosis conditions such as cystic fibrosis (CF) and idiopathic pulmonary fibrosis (IPF).…
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…
Sancilio recently completed enrolling children with sickle cell anemia/disease in a Phase 2 clinical trial to evaluate a new therapy called Altemia (docosahexaenoic acid). The SCOT trial, as it will…
Your hear that? It's the sound of the future knocking on the door! The MIT Technology Review reports about the first-known experiment to edit human embryos in the United States using a…
Trigeminal neuralgia (TN), or extreme, sudden facial pain associated with the 5th cranial nerve, occurs frequently in patients who have received a multiple sclerosis (MS) diagnosis. In a survey with 8,590 respondents,…
The United States FDA just granted Orphan Drug Designation to Napo Pharmaceuticals for Mytesi (crofelemer) in the treatment of short bowel syndrome (SBS). SBS is a rare disorder in which…
Orphan drugs, for those of you who don’t know, are those designated especially for rare diseases. They respond to a public health need, but not necessarily an economic need by…
Sa-Nakht was an ancient Egyptian pharaoh who lived circa 2700 B.C. While there are many tall tales surrounding his reign, one that is especially tall, literally, and not figuratively, is…
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