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…
A new study pinpoints faulty genes as another potential cause behind hemiplegic cerebral palsy. Until now, there was an insufficient amount of information to verify that genetics was part of…
Theravance Biopharma recently proudly announced the results of a successful Phase 2b trial for their gastroparesis treatment, Velusetrag. The results showed that this treatment safely and effectively improved symptoms and normalized…
A biopharmaceutical company based in California called Retrophin recently announced that the first patient has been administered treatment in their phase III study in to test RE-024 (fosmetpantotenate) as a…
Making scientific discoveries must be a lot like trying to map a coastline in the dark. There’s a lot of bumping into things and getting your feet wet in the…
A study by the Sotiria Chest Diseases Hospital in Athens Greece revealed a surprising new link between asthma and bronchiectasis. Visit bronchiecstasisnewstoday.com to read the full article and study results.…
The U.S. FDA just announced that it has accepted Genentech’s supplemental New Drug Application (sNDA) and has granted Priority Review for Zelboraf (vemurafenib) in patients with Erdheim-Chester disease from the…
Mitobridge, Inc., a pharmaceutical company that focuses on improving mitochondrial function, just announced the achievement of a vital milestone in the first-in-human clinical trial of PPAR-delta modulator, MA-0211. This study…
So I don’t think it will surprise anyone to learn that people living with mental illnesses like schizophrenia or bipolar disorder have to put up with a huge ration of…
The Children’s Oncology Group (COG) and the National Cancer Institute (NCI) and have begun collaborating on a new project, to be known as the Pediatric Molecular Analysis for Therapy Choice…
Neuroblastoma, though rare, is the most common type of cancer in children who are five years old or younger. This cancer targets the immature nerve cells in the nerve tissue…
Tissue engineering has a relatively easy concept behind it. First, a patient’s stem cells are grown in the laboratory. Then, they are added to a scaffold material. And ta da!…
BioMarin just announced interim results from a continuing Phase 1/2 study for hemophilia A patients, testing a gene therapy known as BMN 270. Hemophilia A is a rare bleeding disease…
The other day while I was shadowing a neurologist, I encountered a multiple sclerosis (MS) patient. The patient's MS was stable and he was doing quite well on his medications.…
Organ donation is an intensive process to go through. It takes a ton of time to find a match, and even after the donation has taken place, recipients run a…
Soleno Therapeutics, Inc. of Capnia reported in early July of this year that the company plans to start a Phase 3 clinical trial for evaluating diazoxide choline controlled-release (DCCR) in…
Major breakthrough alert! Scientists have developed a method to test the sweat of infants to detect cystic fibrosis (CF). CF is an inherited disorder that causes severe damage to the…
In late July of this year, biopharmaceutical company, Incyte, announced that the very first treatment in a Phase 3 clinical trial for itacitinib had been administered for an acute graft-versus-host…
Genetic engineering has, for a long time, been a highly heated and contested issue. One must weigh the ethical ramifications and risks of engineering human embryos with the huge potential…
We’ve all been there. In the grocery store or at the mall or at the amusement park, when a child starts to act out, much to the chagrin of the…
A recent study found that a specific bacteria may be the distinguishing factor between cardiac sarcoidosis and other conditions that affect the heart. This data suggests that this bacteria may…
They say, “Necessity is the mother of invention.” This maxim goes for everything from nuclear technology (the Manhattan Project was a direct result of World War II) to the Snuggie®…
Parkinson's disease (PD) and amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease) are both neurodegenerative conditions that have no one definitive cause—though there are risk factors linked to…
In late July of this year, the results from a scientific preclinical trial were published in which a research team in Europe discovered initial signs that could pave the way…
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