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It remains unclear why some paediatric tumours appear to have such a low mutation burden. Here, the authors shed light on this paradox by analysing Wilms tumours using high resolution and high ...

Fulcrum had identified inhibitors of p38/ mitogen activated protein kinase (MAPK) as powerful inhibitors of DUX4 expression. DUX4 is the gene that is the root cause of FSHD, a progressive muscle ...

The new drug candidate for FSHD will combine an RNA molecule from miRecule targeting double homeobox 4 (DUX4) – a protein that is mutated in FSHD – with a nanobody developed by Sanofi that ...

1 Department of Pediatrics, Keio University School of Medicine, Tokyo, Japan 2 Department of Neuro-oncology, Institute of Brain Science, Graduate School of Medical Sciences, Nagoya City University, ...