Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) ...

In four groups of boys with DMD (three boys in each group), an escalating ... of antisense oligonucleotides in the modification of SMN2 splicing in patients with SMA are likely to emerge.

In parallel, advances in oligonucleotide technologies for engineering SMN2 pre-mRNA splicing are approaching ... Previous studies with AONs in mouse models of DMD have shown relatively good ...

Entrada Therapeutics has secured the Health Authorities and Ethics Committees' authorisation within the European Union (EU) ...

Early treatment in SMA provides benefits, but children with two SMN2 copies may still experience significant motor delays and disabilities. Infants with two SMN2 copies showed delayed motor ...

Singh, a professor of biomedical science at Iowa State University, led an eight-year project to create a truncated version of the Survival Motor Neuron 2 (SMN2) gene to facilitate quicker ...

DUVYZAT ® (givinostat) is a histone deacetylase (HDAC) inhibitor indicated for the treatment of patients six years of age and older with DMD. Please see the Indication and Important Safety ...