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 ...

In the largest study of its kind globally, UK clinicians and researchers at Royal Manchester Children’s Hospital (RMCH) part of Manchester University NHS Foundation Trust (MFT), and Great Ormond ...

This tells the story of a strong friendship between a young boy with Morquio's syndrome and an older boy who is always bullied because of his size. Adapted from the novel, Freak the Mighty ...