The antisense oligonucleotide works by modulating alternative splicing of the SMN2 exon which is mutated in the disease. This is however "laborious, slow and costly", according to the biopharma.

MENLO PARK, Calif., May 08, 2025 (GLOBE NEWSWIRE) -- PacBio (NASDAQ: PACB) today announced financial results for the quarter ended March 31, 2025. First quarter results: Q1 2025Q1 2024Revenue$37.2 ...

Evaluating MMO proves to be a challenge The 24 children with SMA who were evaluated were < 24 months of age; had SMA confirmed genetically, with 2 or 3 SMN2 copies; and had been administered 1 of ...

The cranial nerves are a set of twelve pairs of nerves that travel to and from the brain. Each has a different function. For example, the olfactory nerve is essential for detecting smells. The ...

who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively.

Digital PCR, which simultaneously detects SMN1 gene defects and the number of SMN2 copies to diagnose spinal muscular atrophy (SMA) in newborn screening programs, matches the accuracy of standard ...

Alternative pre-mRNA splicing is a fundamental mechanism of post-transcriptional gene regulation that creates lineage-specific functional diversity in the proteome. Although heart-specific alternative ...

In this environment, reel splicing kits have evolved from a niche accessory into an essential tool for optimizing SMT operations. Whether running high-mix, low-volume builds or driving 24/7 production ...

Cluster algebras and scattering diagrams appear in diverse areas of mathematics and physics including total positivity, Seiberg duality, statistical physics models, noncommutative geometry, tropical ...