ABSTRACT RNA therapeutics comprise a diverse group of oligonucleotide-based drugs
such as antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and short …

Spinal muscular atrophy is a genetic motor neuron disease that leads to progressive
muscular atrophy and muscle weakness. In December 2016, the Food and Drug …

Background: The antisense oligonucleotide Nusinersen recently became the first approved
drug against spinal muscular atrophy (SMA). It was approved for all ages, albeit the clinical …

Spinal deformities and surgical correction of scoliosis can make intrathecal delivery of
nusinersen very challenging. We aim to evaluate the feasibility and safety of intrathecal …

There are many differences in fluoroscopy-guided lumbar puncture (FG-LP) technique
among radiologists. Even within the same institution, there are a variety of preferences …

Background: Intrathecal administration of nusinersen in adult spinal muscular atrophy (SMA)
patients presents challenges owing to severe scoliosis and previous spinal surgery with …

Background This observational study describes our experience delivering nusinersen
through lumbar puncture with real-time ultrasound guidance in spinal muscular atrophy …

This study describes a new procedure for a safer and easier access for the intrathecal
injection of the recently approved nusinersen therapy in spinal muscular atrophy. This …

Background and purpose The purpose was to report the results of ultrasound‐guided
lumbar puncture for the administration of nusinersen in spinal muscular atrophy (SMA) …

The approval of nusinersen for the treatment of spinal muscular atrophy (SMA) has
significantly changed the natural history of the disease. Nevertheless, scoliosis secondary to …