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

In a second Phase IIIb study, dubbed STRENGTH, OAV101 IT led to stabilization of motor function over 52 weeks in patients who have discontinued treatment with Biogen’s antisense oligonucleotide ...

Risdiplam is an SMN2 pre-mRNA splicing modifier designed to increase and sustain SMN protein levels centrally and peripherally. It's one of three drugs approved for SMA, the others being ...

Versatile export options: users can print out splice diagrams in various formats or export them to other CAD systems via VSD format, ensuring easy integration with existing workflows.

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 ...

Johns Hopkins researchers have developed a powerful new AI tool called Splam that can identify where splicing occurs in genes—an advance that could help scientists analyze genetic data with greater ...

The modulation of RNA splicing by small molecules has emerged as a promising strategy for treating pathogenic infections, human genetic diseases, and cancer; however, the principles by which ...

An almost-microscopic creature that's sturdy enough to survive the ravages of space may hold the key to human longevity, scientists have found in new research. In a new study led by the University ...