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 ...

She’s one in 200,000. Diagnosed with a rare condition In February 2024, when she was five, Liliana was diagnosed with Morquio syndrome — also known as mucopolysaccharidosis type IV — a rare ...