Potential ASO Therapy Treats SMA in Mice With Evident Symptoms

Potential ASO Therapy Treats SMA in Mice With Evident Symptoms

314813

Potential ASO Therapy Treats SMA in Mice With Evident Symptoms

A new and noninvasive type of antisense oligonucleotide (ASO) therapy effectively treated spinal muscular atrophy (SMA) in a mouse model with established symptoms, a study reported. Since symptomatic and late-onset SMA patients are the most difficult to treat, “our [therapy] should be considered a great improvement from a clinical perspective,” its scientists wrote. “We believe strongly in the feasibility of clinical translation and the utility” of this potential treatment “even in symptomatic SMA patients.” The…

You must be logged in to read/download the full post.