In this issue:
Reducing false-positives in NBS
Autologous genome-edited hematopoietic stem cells for MPS I
DNL310 (brain-penetrant ERT) in MPS II
Somatic outcomes of DNL310
Pabinafusp alfa for neuronopathic MPS II
Pabinafusp alfa & retinal disease in MPS II mice
Amniotic fluid GAGs from in-utero ERT-treated MPS
First-in-human trial of genetically engineered B cells
Autologous B cell therapy for Fabry disease
B cell medicine for Neimann-Pick disease
Autologous HSCGT for MPS IIIA
Diagnosis of Fabry disease in the UK
A novel high-throughput screening assay
NfL levels & MPS II outcomes
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