Niemann-Pick B - RS - 5th infusion.
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PDF) Defects in sarcolemma repair and skeletal muscle function after injury in a mouse model of Niemann-Pick type A/B disease
JCM, Free Full-Text
Natural history and management of liver dysfunction in lysosomal storage disorders
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Frontiers Potential Disease-Modifying Effects of Lithium Carbonate in Niemann-Pick Disease, Type C1
Current Clinical Applications of In Vivo Gene Therapy with AAVs: Molecular Therapy
Interstitial lung disease in lysosomal storage disorders
Frontiers Expert opinion on patient journey, diagnosis and clinical monitoring in acid sphingomyelinase deficiency in Turkey: a pediatric metabolic disease specialist's perspective
Niemann–Pick disease - Wikipedia
PDF) Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann–Pick disease type B (acid sphingomyelinase deficiency)
(PDF) Niemann-Pick Disease: An Underdiagnosed Lysosomal Storage Disorder
Niemann-Pick C1 expression is not regulated by the amount of cholesterol flowing through cells in the mouse - ScienceDirect
IJMS, Free Full-Text
Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals
Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion - ScienceDirect
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