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Neutral Lipid Storage Disease
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Neutral Lipid Storage Disease trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Neutral Lipid Storage Disease trials may be worth asking aboutNorth America
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This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participant…
This study aims to characterize the pathophysiological mechanisms of 21 different metabolic myopathies. The study will focus on exercise capacity and the metabo…