KidsHealth1 年
Morquio A Syndrome
What Is Morquio A Syndrome? Morquio A syndrome (mucopolysaccharidosis IVA; MPS IVA) is one of a group of disorders called mucopolysaccharidosis (MPS). In MPS, there’s either not enough of the enzyme ...
KidsHealth1 年
Morquio A Syndrome
What Is Morquio A Syndrome? Morquio A syndrome (mucopolysaccharidosis IVA; MPS IVA) is one of a group of disorders called mucopolysaccharidosis (MPS) . In MPS, there’s either not enough of the enzyme ...
pharmaphorum2 年
NICE firms up NHS use of Vimizim for Morquio A syndrome
In 2015, NICE said that BioMarin's Vimizim could be used to treat patients with the ultra-rare disease Morquio A syndrome ... works by replacing a missing enzyme that is needed to break down ...