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Evidence-based recommendations on sebelipase alfa (Kanuma) for long-term enzyme replacement therapy in Wolman disease (rapidly progressive lysosomal acid lipase deficiency) in people aged 2 years and under when treatment starts.
NICE is unable to make a recommendation on sebelipase alfa (Kanuma) for treating lysosomal lipase deficiency that is not Wolman disease in adults. The Wolman disease population was evaluated separately in NICE highly specialised technology guidance on sebelipase alfa (HST30). The cost effectiveness of the remaining non-Wolman population has not been demonstrated at this stage.
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pemetrexed 6 June 2007 TBC Lysosomal acid lipase deficiency - sebelipase alfa 25 April 2017 TA068 Macular degeneration (age-related) -...