Search Results for: Antisense oligonucleotides

Antisense Oligonucleotides (RaNA)

Antisense oligonucleotides (ASOs) are small molecules that can bind to RNA to change how it is spliced. It is a potential therapy for SMA, as customizing ASOs may change ... Read more

Experimental Therapies for SMA

Spinal muscular atrophy (SMA) is an autosomal recessive genetic disorder, characterized by progressive muscle weakness caused by a loss of specialized nerve cells (motor neurons) in the spinal cord ... Read more

ASOs to Treat SMA

Antisense oligonucleotides (ASOs) have the potential to treat most diseases that involve altered mRNA expression due to gene mutations, such as spinal muscular atrophy (SMA). ASOs are small synthetic ... Read more