The SMN2 splicing modifiers penetrated into all mouse tissues tested, including brain, spinal cord, and muscle, and thus improved SMN2 RNA splicing to increase SMN protein production in these ...

The study used chemical screening and optimization to identify orally available small molecules that selectively alter the splicing of the SMN2 pre-mRNA to produce stable full-length SMN protein.

Severe SMA mice tend to be small in relation to wild-type mice, and SC administration of ASO-10-27 has a particularly marked effect on SMN2 splicing in the liver, which contributes about 75% of ...

SOUTH PLAINFIELD, N.J., Aug. 7, 2014 /PRNewswire/ -- Today the journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of ...

The pre-mRNA SMN2 splicing modifier risdiplam was administered orally to 41 infants with type 1 spinal muscular atrophy. After 12 months of treatment, 12 infants were able to sit without support ...