Antisense technology is beginning to deliver on the broad promise of the technology. Ten
RNA-targeted drugs including eight single-strand antisense drugs (ASOs) and two double …

A total of 378 novel drugs and 27 biosimilars approved by the US Food and Drug
Administration (FDA) between 2010 and 2019 were evaluated according to approval …

Background & Aims Spinal muscular atrophy (SMA) is an autosomal recessive, childhood-
onset motor neuron disease. Onasemnogene abeparvovec (OA) is a gene therapy designed …

Splice-switching oligonucleotides (SSOs) are short, synthetic, antisense, modified nucleic
acids that base-pair with a pre-mRNA and disrupt the normal splicing repertoire of the …

Spinal muscular atrophy type 1 (SMA1) is a debilitating neurodegenerative disease resulting
from survival motor neuron 1 gene (SMN1) deletion/mutation. Onasemnogene abeparvovec …

Recent advances in the treatment of spinal muscular atrophy (SMA) have dramatically
altered prognosis. Rather than a rapidly lethal disease, SMA type 1, the most severe form …

Spinal muscular atrophy (SMA) is a recessive disorder caused by a mutation in the survival
motor neuron 1 gene (SMN 1); it affects 1 in 11 000 newborn infants. The most severe and …

Background: Spinal muscular atrophy type 1 (SMA1) is the leading genetic cause of infant
mortality for which therapies, including AVXS-101 (onasemnogene abeparvovec …

Spinal muscular atrophy (SMA) is a heritable neuromuscular disorder that causes
degeneration of the alpha motor neurons from anterior horn cells in the spinal cord, which …

Copy number variants (CNVs) were the subject of extensive research in the past years. They
are common features of the human genome that play an important role in evolution …