RHO and proximal spinal muscular atrophy: In addition, an ASO-based allele-specific mRNA knockdown/degradation approach for dominantly inherited RP caused by pathogenic variants in RHO is underway in a clinical trial (NCT04123626), as are two different approved treatments for spinal muscular atrophy and Duchenne muscular dystrophy based on ASOs that modulate the splicing process.98