WOODBRIDGE – A fundraiser to benefit brave 4-year-old township resident Scarlett Griffith and heighten awareness about the ...

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 ...

The decision has also been welcomed by charity the MPS Society, which represents people with the ultra-rare condition, mucopolysaccharidosis type IVa (MPS IVa), or Morquio. While the decision has ...

Gracie Mellalieu, 18, is one of just 3,000 people across the world who has Morquio Syndrome which has many effects including ...

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 ...