According to a story from PR Newswire, the international drug developer Eli Lilly and Company recently announced the results of a Phase 3 clinical trial testing the company's drug LARTRUVO…
According to an article published by Washington University of Medicine in St. Louis, scientists appear to have succeeded in slowing axon destruction in mice through standard gene therapy. Though an…
A concerned Texas mom finally has answers about the cause of her son’s sensory-processing problems, thanks to a special collaboration among her patient-advocacy group and the lab of Scripps Research…
The Florey Institute of Neuroscience (FIN), one of the world’s largest and most respected brain research centers, recently announced trial results for the drug CuATSM, developed by FIN, the School…
David Radford, a 63-year-old resident of Oxford, UK, and a liver cancer survivor, said his surgeon attributes his successful liver transplant to the new normothermic perfusion machine. As reported recently…
According to a story from Medical Xpress, a recent study found that people who survived Hodgkin lymphoma during their childhood were at a greater risk of developing another type of…
Hope everyone is having a wonderful Wednesday! The weather where we are is freezing this week-- but that's a perfect excuse to sit down with a few of our top…
Danon disease is a rare condition caused by a mutated LAMP-2 gene. This gene has three different forms- LAMP-2A, 2B, and 2C. Depending in which variant is mutated, individuals with…
According to a story from BioPortfolio, the biotechnology company Vertex Pharmaceuticals recently announced that the European Commission has approved a label extension for the company's cystic fibrosis drug Orkambi. Under…
aTTP aTTP, or acquired thrombotic thrombocytopenia purpura, is an immune condition which results in severe Thrombocytopenia. Thrombocytopenia means the body doesn't have enough platelets in the blood. In aTTP, it's caused…
According to a story from news-medical.net, a recent study has a found that a hidden disease called hemochromatosis could be a major risk factor for several different joint diseases and…
Pharnext SA, a biopharmaceutical company, recently announced results in a PLOS ONE article entitled "Early short-term PXT3003 combinational therapy delays disease onset in a transgenic rat model of Charcot-Marie-Tooth disease 1A…
According to a publication from Stat, scientists have high hopes about "virtual repurposing" cutting drug production costs. It could also potentially mean more treatments on the market faster. Virtual Repurposing…
According to a report from ITV, a five-year-old boy from southern England has made the trip across the Bay of Biscay to Barcelona, Spain, where he is receiving an experimental…
Vogmask is a San Francisco, California based company founded in 2011. They create stylish facial masks to protect wearers from inhalation of particulate and allergens. These types of masks can…
This is week three of this season of The Bachelor on ABC, and week three of a patient-related subject highlighted on the show (Colton's CF foundation, Caelynn's childhood encephalitis)-- so I'm…
Read the first part of Lisa's story here. Lisa’s two direct and indirect bypass surgeries were successful. She now had seven times more blood flow to her brain than she…
UPI Health News recently reported that a study, published in the New England Journal of Medicine, showed that fecal microbiota transplantation resulted in a 32 percent remission rate in patients…
According to a story from dailymail.co.uk, the story of Cayla Jones is an example of how a serious rare diagnosis can suddenly strike out of nowhere, often from symptoms that…
Bontulinum Neurotoxins (BoNTs) are a protein that are used to treat a wide array of conditions including cervical dystonia, blepharospasm, neurogenic detrusor overactivity, and sialorrhea to name a few. These proteins…
According to an article published by R&D, scientists at Harvard have identified a new genetic clue that they suspect might be linked to the development of amyotrophic lateral sclerosis (ALS).…
The development of gene therapy as a treatment for rare and life-threatening conditions is a quickly growing area of scientific exploration. As more drug developers have begun investing in this…
According to an article from Global Banking & Financing Review, the California-based pharmaceutical company Exelixis, Inc. has announced the FDA's approval of their drug Cabometyx (cabozantinib) for patients previously diagnosed…
Cystinosis Cystinosis is a rare disease which is caused by a buildup of cystine in the body's cells. This buildup impacts various organs including the brain, eyes, liver, kidneys, and muscles.…
According to a story from Biotecnika, Penn Medicine & Inovio Pharmaceuticals Inc. have recently announced that the US Food and Drug Administration (FDA) has signaled its approval for the first…
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