In this edition:
Small activating RNAs for monogenic rare diseases
Development of consensus guidelines for Sanfilippo syndrome
Spectrum of podocyte injury in late-onset Fabry disease
Deep learning tool for measurement of foot process width
Plasma lyso-sphingomyelin: biomarker for acid sphingomyelinase deficiency
Long-term growth in acid sphingomyelinase deficiency treated with olipudase alfa
Defect-free graphene enhances enzyme delivery to fibroblasts
Imiglucerase in paediatric Gaucher disease types 1 and 3
Translational model of neuropathic Gaucher disease
Progranulin-derived biologic for neuronopathic Gaucher disease
Neurodegeneration in progranulin knockout mice
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