Gio was born with a rare genetic condition called Morquio A syndrome. It affected the growth of his bones and significantly shortened his life expectancy. "They told us 10, 12 years, probably, and he ...

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 ...

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 being ...