Keyphrases
Abnormal Liver Chemistries
11%
Acid Sphingomyelinase Deficiency
100%
Adult Patients
11%
Cirrhosis
11%
Clinical Development
11%
Disease Biomarker
11%
Disease Burden
11%
Dose Escalation
11%
Electron Microscopy
11%
Enzyme Replacement Therapy
11%
Escalating Dose
11%
Fibrosis
22%
Hepatocytes
11%
Histomorphometric Analysis
11%
Inflammation
11%
Kupffer Cells
11%
Lipid Profile
100%
Liver Biopsy
44%
Liver Function Tests
22%
Liver Involvement
11%
Liver Volume
22%
Lysosomal Storage Disease
11%
Morphometry
11%
Neurological Manifestations
11%
Neuronopathy
11%
Niemann-Pick Disease Type A
11%
Olipudase Alfa
100%
Patient Dose
11%
Pharmacodynamic Biomarker
22%
Phase 1b Study
11%
Recombinant Human
11%
Relative Reduction
11%
Sphingomyelin
100%
Tolerability
11%
Medicine and Dentistry
Acid Sphingomyelinase
100%
Biological Marker
22%
Biopsy Technique
22%
Disease Burden
11%
Electron Microscopy
11%
Enzyme Replacement Therapy
11%
Fibrosis
22%
Hepatocyte
11%
Hepatomegaly
11%
Kupffer Cell
11%
Liver Biopsy
44%
Liver Cirrhosis
11%
Liver Function Test
22%
Lysosomal Storage Disease
11%
Morphometrics
22%
Neurologic Finding
11%
Niemann-Pick Disease
11%
Olipudase Alfa
100%
Pharmacodynamics
22%
Recombinant
11%
Sphingomyelin
100%
Storage Disease
11%
Pharmacology, Toxicology and Pharmaceutical Science
Biological Marker
22%
Disease
11%
Electron Microscopy
11%
Fibrosis
22%
Hepatomegaly
11%
Inflammation
11%
Liver Cirrhosis
11%
Lysosome Storage Disease
11%
Niemann Pick Disease
11%
Olipudase Alfa
100%
Pharmacodynamics
22%
Recombinant
11%
Replacement Therapy
11%
Sphingomyelin
100%
Sphingomyelin Phosphodiesterase
100%
Storage Disease
11%
Tolerability
11%