Love them, hate them, or love to hate them - celebrities are a big part of our culture. They sell us clothes, perfumes, lifestyles - and some we even elect…
As the United States Senate gears up to vote on whether to pass their version of the Republican-crafted tax reform legislation (the House of Representatives has already passed their version),…
You're familiar with the formula of the rom-com: Boy meets girl, girl falls in love boy, boy faces obstacle, girl almost loses boy - but eventually big kiss and happily…
Earlier this month, the FDA approved Zelboraf (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. The final results indicated a response rate of 54.5%. This is the first FDA-approved treatment…
Earlier this month, The Lancet Neurology published results from its PATH study (Polyneuropathy And Treatment with Hizentra), which showed efficacy in treating people with chronic inflammatory demyelinating polyneuropathy (CIDP). Great news and hope…
Big news for those with hemophilia! On Thursday, the FDA approved Hemlibra (manufactured by Genentech), a weekly self-injection for hemophilia A patients who have developed resistance to standard medicines for preventing…
"I believe there is a solution." Those are billionaire mogul Bill Gates's words regarding the future of Alzheimer's. "Any type of treatment would be a huge advance from where we…
Enfermedad crónica afecta para siempre la perspectiva de la vida de un paciente. A veces, esa perspectiva depende de cómo el paciente se siente físicamente en ese momento. Después de…
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?…
Si usted o alguien que usted conoce tiene síndrome de Ehlers-Danlos (EDS), usted probablemente sabe mucho sobre él, que te hace parte de un grupo muy pequeño. No mucha gente…
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…
A California judge threw out a $417 million verdict against Johnson & Johnson in a lawsuit by Eva Echeverria - who claimed she developed ovarian cancer after using Johnson &…
The Bank of America building in Dallas, Texas will light itself up in turquoise on Saturday October 28th to show support for Dysautonomia Awareness Month. Everything's bigger in Texas, including…
A team of researchers at the Cincinnati Children’s Hospital Medical Center has developed a method to test for Gaucher disease using dried blood samples - marking a big breakthrough for the Gaucher…
Scientists at the University of Georgia have identified a rare type of glycosylation (more on what that is in a second) which plays a major role in the function of…
It's been a tough year for the United States Congress and healthcare legislation - you might have heard, they failed to repeal and replace the Affordable Care Act nor have…
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…
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…
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…
Pharma giant Novartis's new cancer drug tisagenlecleucel passed a critical test by winning the support of a federal advisory panel - setting it up for final FDA approval in September.…
According to a recent study in Science Daily, scientists have found evidence that one treatment drug's effect on the cells for a specific condition could actually be applied to several…
Leave it to a sports award show to recognize what a real fighter looks like. ESPN's annual award show (the ESPYs) honored 15 year-old Jarrius Robertson last week with the…
Great news for our friends in the the Gaucher community! The FDA and its counterpart across the pond, the European Medicines Agency (EMA), have outlined a new strategy to spur…
You heard that right, Starbucks lovers! According to two different studies - one from the US and one from Europe - drinking anywhere from one to three cups of coffee…
A new study detailed in Nature, involving synthetically replicating certain cell activity has been hailed as a major breakthrough in developing therapies for many neurological disease – Parkinson’s chiefly among…
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