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 ...

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.

Gracie Mellalieu, 18, is one of just 3,000 people across the world who has Morquio Syndrome which has many effects including on her mobility. Now she is championing the innovative scheme that’s ...