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 ...

Gio passed away at the age of 5, but his family and medical team remember the joy he brought into their lives.

Gio, as he was known, was diagnosed with Morquio Syndrome, a rare and progressive birth defect that impacts the body's ability to function over time. Donnelly only worked with him for six months.

But for Dr. Montaño, it’s more than just research—her work on Morquio A syndrome, a rare inherited birth defect that impacts a child’s appearance, organ function, and physical abilities, is personal ...

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 ...

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 ...