FDA OK's Vimizim; BioMarin wins 1st rare peds disease voucher
This article was originally published in Scrip
Two weeks early, the FDA on 14 February approved BioMarin Pharmaceutical's enzyme replacement treatment (ERT) Vimizim (elosulfase alfa) for a rare lysosomal storage disorder, known as Mucopolysaccharidosis Type IVA (MPS IVA), which causes progressive skeletal dysplasia, including joint deformities and contractures, short stature and spinal cord compression, and reduced pulmonary function, valvular heart disease, hearing loss, cataracts and corneal clouding.
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