Burlington resident Candice Russell has to hold her six-year-old daughter, Lilliana, down so a nurse can administer her ...

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.

For the first time, Hospital Kuala Lumpur (HKL) is proudly hosting the annual Rare Disease Day 2025 celebration to raise ...

Some forms, like MPS I (Hurler syndrome) and MPS II, also involve severe neurological decline, while others, such as MPS IV (Morquio syndrome), primarily affect the skeletal system without ...