资讯

Teenager with one of world's rarest conditions opening doors for others with disabilities
Gracie Mellalieu, 18, is one of just 3,000 people across the world who has Morquio Syndrome which has many effects including ...
pharmaphorum3 年
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 ... collected clinical data from 69 people treated with Vimizim ...