Full Patient Enrollment in Study Expected by Year-End 2019 LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Feb. 04, 2019 -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for…
Multiple Myeloma Multiple Myeloma is a rare form of cancer that develops in the body's plasma cells. These cells are normally responsible for helping the body fight infection. The accumulation…
According to a story from Genetic Obesity News, the unusual case of a female Bardet-Biedl syndrome patient who also had endometrial cancer is causing researchers to wonder if the two…
Rett Syndrome Rett syndrome is a rare neurodevelopmental disorder. It affects females disproportionately, at a far greater rate than males. It causes cognitive, autonomic, and neurological dysfunction as well as a loss of…
Hereditary angioedema (HAE) is a rare condition which causes severe swelling in various areas of the body. Takhzyro has been approved as a treatment for this disease. The drug was created…
Happy Wednesday! While it's felt like January was going to last forever, we're almost through the first month of 2019. As we gear up for February, we want to reflect…
Epidermolysis Bullosa Epidermolysis Bullosa (EB) refers to a group of rare conditions which are characterized by fragile skin. Basically, the disease is caused by separation between the layers of the skin.…
According to an article from Pharmaceutical Daily, the Seattle-based drug company Sound Pharmaceuticals is set to soon begin a phase 2 clinical study for a cystic fibrosis (CF) drug candidate…
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