UK NICE recommends use of Vimizim in mucopolysaccharidosis type 4A.
The National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending Vimizim (elosulfase alfa), from BioMarin, for routine use in the NHS, treating mucopolysaccharidosis type 4A (MPS 4A) – a rare metabolic condition that primarily affects the skeleton.
This is the first disease modifying treatment recommended by NICE for routine NHS use.
MPS 4A (also known as Morquio A syndrome) is an autosomal recessive genetic condition which causes growth delays, a prominent lower face, hip dislocation and arthritis, concern for a spine abnormality and an abnormally short neck.
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