Early diagnosis of infantile-onset lysosomal acid lipase deficiency in the advent of available enzyme replacement therapy

Overview of attention for article published in Orphanet Journal of Rare Diseases, August 2019

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Fiona White hi-lighting this paper from Cohen et al, https://t.co/O63mUIVfBV The growth data shared comes from the supplementary figure. #BIMDG2021 https://t.co/mZDDNEGlvo

Early diagnosis of infantile-onset lysosomal acid lipase deficiency in the advent of available enzyme replacement therapy https://t.co/xQPYe0pLJO