According to a story from Market Screener, the biotechnology company BioMarin Pharmaceutical Inc. recently announced that the Chinese government has approved the company's drug elosulfase alfa (marketed at Vimizim) as…
Researchers at the Massachusetts General Hospital Research Institute and the Georgia Institute of Technology are investigating a medication currently approved to treat a type of multiple sclerosis as a possible…
12-year-old Cezy Fosca lives in Kilkenny City and suffers from Morquio syndrome. Her family waited for months on approval for her treatment, after she lost access to it. Finally, the…
Yesterday, a group of experts presented "Changing lives: Long-term outcomes of MPS IVA & VI patients" at the Satellite Symposium sponsored by BioMarin. The program began with insights on the…
Back in 2014, the FDA gave the green light for eight brand new drugs for orphan diseases. Among the pack was BioMarin Pharmaceutical's very own VimiZim. Weird name right? Well,…
Recent findings from research done at the University of Pennslyvania indicate a new biomarker associated with a group of rare metabolic diseases called mucopolysaccharidoses (MPS). A biomarker is a measurable…
We all have those memories of Christmas time when we wanted that one toy so badly. We would have gladly given an arm or a finger to get that gaming…
As humanity collectively slinks into the cold, dark days of winter, we could all use something to warm us up… or at least bring a small slice of sunshine. Well,…
What would you do if you had a medical condition that required treatment that costs thousands of dollars a week? If nothing else, you’d reach your deductible in the first…
I have a new hero, an Irishman who’s not only gotten some great press and notoriety, this guy has struck gold in the hearts of the Morquio syndrome community! In…
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