In this review:
Biomarker differentiating neuronopathic forms of MPS I and MPS II
Ex-vivo autologous stem cell gene therapy for MPS II
Brain-penetrant ERT for MPS II
Arimoclomol reduces lipid biomarkers in NPC
Incidental lysosomal disease diagnosis by genetic testing
Paediatric experience of Fabry patients
Age and previous diagnoses in UK Fabry disease patients
In utero ERT for Pompe disease
Newborn Pompe disease screening experience in Minnesota
Avalglucosidase-α in IOPD
Please login below to download this issue (PDF)