18 February 2016 - The draft guidance recommends that the research should be designed to generate robust evidence about the costs and benefits of long-term treatment with sebelipase alfa compared with shorter term treatment (‘bridging therapy’) followed by blood and marrow transplantation (also known as haematopoietic stem cell transplantation) in people diagnosed with rapidly progressive LAL deficiency before they were 6 months old.

For more details, go to: http://www.nice.org.uk/news/press-and-media/nice-calls-for-more-research-into-promising-new-drug-to-treat-inherited-rare-enzyme-deficiency