Antisense technology is now beginning to deliver on its promise to treat diseases by
targeting RNA. Nine single-stranded antisense oligonucleotide (ASO) drugs representing …

RNA-binding proteins (RBPs) are critical effectors of gene expression, and as such their
malfunction underlies the origin of many diseases. RBPs can recognize hundreds of …

Abstract SPR1NT (NCT03505099) was a Phase III, multicenter, single-arm study to
investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic …

Spinal muscular atrophy (SMA) is a neurodegenerative disorder caused by mutations in
SMN1 (encoding survival motor neuron protein (SMN)). Reduced expression of SMN leads …

Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular
atrophy (SMA) type 2. SPR1NT (NCT03505099), a Phase III, multicenter, single-arm trial …

Importance This ongoing study assesses long-term safety and durability of response in
infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene …

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …

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 …

Despite extensive research, amyotrophic lateral sclerosis (ALS) remains a progressive and
invariably fatal neurodegenerative disease. Limited knowledge of the underlying causes of …

Oligonucleotides (ONs) comprise a rapidly growing class of therapeutics. In recent years, the
list of FDA-approved ON therapies has rapidly expanded. ONs are small (15–30 bp) …