RWD shakes confidence in elusulfase alfa for MPS 4A

Written by Darcy Hodge, Editor

elusulfase alfa

Draft guidance published by NICE (London, UK) has not recommended the use of elusulfase alfa, otherwise known as Vimizin®, for mucopolysaccharidosis type 4A (MPS 4A) on the NHS. The decision comes after new research evidence was collected, including real-world data (RWD). MPS 4A is a rare condition that shortens the life of individuals, by lacking the N-acetylgalactosamine-6-sulfatase enzyme. Consequently, glycosaminoglycans accumulate in tissues and organs, leading to skeletal abnormalities, vision loss and a gradually increasing reliance on wheelchairs. Life expectancy for individuals with MPS 4A is reduced; on average, to around 25 years old. Since the treatment has been available...

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