For many rare diseases, gene therapy, not traditional therapeutics, is perhaps the only means to significant relief and cure. However, the path to gene therapy has been strewn with obstacles…
Gracie Mellalieu, a twelve-year-old girl from Wales, is excited to headline the Christmas concert at St. Asaph Cathedral. This accomplishment comes after Gracie gained attention for a thank you song…
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…
According to a story from the Irish Mirror, eleven year old Cezy Fosca, who has a rare condition called Morquio syndrome, has lost access to the only drug available that…
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…
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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