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Six-year-old NL girl battling rare genetic condition
Burlington resident Candice Russell has to hold her six-year-old daughter, Lilliana, down so a nurse can administer her ...
pharmaphorum2y
NICE firms up NHS use of Vimizim for Morquio A syndrome
In 2015, NICE said that BioMarin's Vimizim could be used to treat patients with the ultra-rare disease Morquio A syndrome, while real-world data was collected on its efficacy. Now – almost seven ...