The Spanish National Cancer Research Centre (CNIO) announced the discovery of a protein called RanBP6 as a regulator of epidural growth factor receptor (EGFR). The study, as originally printed in…
On December 11, Alexion Pharmaceuticals, Inc. released dose-ranging data from a recent clinical study involving treatment with ALXN1210, which is an investigational long-acting C5 complement inhibitor meant to treat sufferers…
Latest research from a University of Chicago team found that dynamic contrast-enhanced MRIs (DCE-MRIs) were much better in early detection of breast cancer than annual mammograms for patients with high-risk…
Caplacizumab is a medicine that has been developed to treat acquired thrombotic thrombocytopenic purpura (TTP). TTP causes serious symptoms. such as thrombocytopenia, a condition in which a patient doesn't have enough platelets. A…
Hereditary hemochromatosis is a genetic form of a condition in which the body builds up too much iron. To read more about hemochromatosis, click here. La Jolla Pharmaceutical Company has…
Everyone with Facebook surely remembers when people were posting videos of themselves having ice cold water dumped on their heads. But, does everyone remember what it was for? The Ice…
Earlier this month, researchers at the University of Montreal found an encouraging way to improve treatments to cystic fibrosis patients. They discovered key molecules, known as quorum-sensing inhibitors, could be…
REGENXBIO Inc., a pharamaceutical company specializing in gene therapy, just shared that it launched a Investigational New Drug application to conduct Phase I/II trials on the new therapy they're developing,…
A recent study at Sanford Burnham Prebys Medical casts a ray of hope in treating multiple hereditary exostoses (MHE). MHE is a rare genetic disorder characterized by multiple noncancerous bone…
Miracle workers at Queens University are working on a revolutionary method to treat patients with Lyme disease. Lyme disease is an infectious disease that springs from a tick bite's bacteria.…
Irish drugmaker, Shire, announced earlier this month it will bring new genetic disorder drugs to India, stepping up its worldwide presence. Shire is unique in that it's one of the…
Neuropsychiatric disorders can be some of the most challenging conditions to understand. Mechanically, socially, and biologically, it is often difficult to make sense of their innermost workings. Recent research, however,…
Pharmaceutical company and gene therapy leader, uniQure, published promising data from their current phase I/II trial testing out a gene therapy called AMT-060 in patients with serious cases of hemophilia B.…
A new study may lead to new cures and treatments for Marfan syndrome. Researchers at Johns Hopkins have made great progress testing in mice. Follow the original story here. On…
On this season of America's Got Talent, the world met Christian Guardino. I was one of millions who heard his beautiful singing voice as he belted out his song onstage.…
Patients diagnosed with Duchenne muscular dystrophy (DMD) have always lived with a grim prognosis. Many don't make it into their 20s and face debilitating symptoms years earlier-- a dark reminder…
Jay and Amy Granzow are among the many worried parents fearing the GOP tax plan, because of the threat it brings to orphan drug research. The Manhattan Beach couple fear…
Robbie Edwards has a rare disease, one so rare that she's one of only 17 people in the world with it. She suffers from hereditary spastic paraplegia which represents a…
As a scientist, Mary-Claire King has had a remarkable career related to her study of BRCA genes. She’s received a Lasker Award and has been recognized by President Obama with…
Not all bacteria are created equal, but all play a role in sustaining--or breaking down--health. And when you're talking about children with cystic fibrosis (CF), a lung disease that impairs…
The internet has been trying to figure out the caffeine question for years: is it good for us? Is it bad for us? Will it stunt our growth? Will it prevent…
In the UK, Newcastle University and Pfizer are collaborating on a $39.9 million-dollar funded project aimed at developing greater tools for testing patients at the most risk for non-alcoholic fatty…
Although Parkinson's disease (PD) affects men and women equally, researchers have discovered an area of disparity: care giving. A study published this month in Neurology by researchers at the Perelman School of Medicine…
The FDA has granted a special designation for a drug company developing a gene therapy for the treatment of leber congenital amaurosis (LCA). MeiraGTx received rare pediatric disease designation for its…
36-year-old Steve Pete has never felt physical pain in his life. While that may sound appealing, it comes with some serious downsides, such as literally chewing off his own tongue…
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